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Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy

Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy

FDA Approval Of Gene Therapies For Sickle Cell Disease: Q&A With NHLBI Director Dr. Gary Gibbons And NHLBI’s Division Of Blood Diseases And Resources Director Dr. Julie Panepinto

FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto

An Ethical And Financial Obligation For Sickle Cell Disease Gene Therapy In The United States

An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States

SCDAA Teams With MedicAlert Foundation To Improve Emergency Outcomes During Sickle Cell Crises

SCDAA Teams with MedicAlert Foundation to Improve Emergency Outcomes During Sickle Cell Crises

$100,000 Grant Allows National Expansion In Expert Care Of Sickle Cell Patients

$100,000 Grant Allows National Expansion in Expert Care of Sickle Cell Patients

SCDAA Partners With Phi Beta Sigma Fraternity

SCDAA Partners With Phi Beta Sigma Fraternity

Sickle Cell Disease Association Of America, Inc. And Hemanext® Inc. Form New Strategic Partnership

Sickle Cell Disease Association of America, Inc. and Hemanext® Inc. Form New Strategic Partnership

Sickle Cell Disease: After Years Of Neglect Some Promise For Sufferers

Sickle Cell Disease: After Years of Neglect Some Promise for Sufferers

Novel Sickle Cell Drug Causes Radical Results In End Of Life Patients

Novel Sickle Cell Drug Causes Radical Results in End of Life Patients

A New Sickle Cell Disease Drug Holds Much Promise But Most Sufferers Won’t Be Able To Afford It

A new Sickle Cell Disease Drug Holds Much Promise but Most Sufferers Won’t be Able to Afford it

FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease

Global Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities With ADDMEDICA And Bristol-Myers Squibb | Technavio

Global Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squibb | Technavio

FDA Approves First Targeted Therapy to Treat Patients with Painful Complication of Sickle Cell Disease

Pfizer Executive Outlines Vision For Treating Sickle Cell Disease, Other Rare Diseases

Pfizer Executive Outlines Vision for Treating Sickle Cell Disease, Other Rare Diseases

GBT launches ACCEL grants program to improve access to care for people with Sickle cell disease

Emmaus Life Sciences Launches Its Commercial Co-Payment Assistance Program for Endari™

Ghana Launches Partnership With Novartis To Improve Diagnosis And Treatment Of People With Sickle Cell Disease

Ghana Launches Partnership With Novartis to Improve Diagnosis and Treatment of People With Sickle Cell Disease

FDA Agrees Accelerated Approval Pathway For GBT’s Voxelotor

FDA agrees accelerated approval pathway for GBT’s voxelotor

Sickle Cell Foundation Nigeria, Rhieos Develop 1st Multi-SCD Registry In Nigeria

Sickle Cell Foundation Nigeria, Rhieos Develop 1st Multi-SCD Registry in Nigeria

Emmaus, A Leader In Sickle Cell Disease Treatment, Signs Agreement With Cardinal Health To Solidify Distribution Network For Endari™ (L-glutamine Oral Powder)

Emmaus, a leader in sickle cell disease treatment, signs agreement with Cardinal Health to solidify distribution network for Endari™ (L-glutamine oral powder)

Siklos (hydroxyurea) Now Available Through ProCare Pharmacy Care’s Siklos At Home Program

Siklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home program

Doris Duke Charitable Foundation Awards Grant To Critical Path Institute

Doris Duke Charitable Foundation awards grant to Critical Path Institute

Emmaus, A Leader In Sickle Cell Disease Treatment, Signs Agreement With McKesson To Expand Distribution Network For Endari™ (L-glutamine Oral Powder)

Emmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for Endari™ (L-glutamine oral powder)

GBT Expands Sickle Cell Disease Pipeline With Worldwide Licensing Agreement For Inclacumab For The Treatment Of Vaso-occlusive Crisis

GBT expands sickle cell disease pipeline with worldwide licensing agreement for inclacumab for the treatment of vaso-occlusive crisis

The Links, Incorporated Presents Million-dollar Grant To St. Jude’s Hospital

The Links, Incorporated presents million-dollar grant to St. Jude’s Hospital

MaxCyte And U.S. National Institutes Of Health’s National Heart, Lung, And Blood Institute Enter Cooperative Research & Development Agreement For Sickle Cell Disease

MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative research & development agreement for Sickle Cell Disease

Novo Nordisk Obtains Licence For Sickle Cell Disease Program

Novo Nordisk obtains licence for sickle cell disease program

Emmaus Life Sciences Receives Validation From European Medicines Agency On Marketing Authorization Application For Sickle Cell Disease Treatment Xyndari (oral Glutamine)

Emmaus Life Sciences Receives Validation From European Medicines Agency on Marketing Authorization Application for Sickle Cell Disease Treatment Xyndari (oral glutamine)

California Institute For Regenerative Medicine Awards City Of Hope $5.74 Million For Severe Sickle Cell Disease Clinical Trial

California Institute For Regenerative Medicine Awards City Of Hope $5.74 Million For Severe Sickle Cell Disease Clinical Trial

University Of Illinois At Chicago Awarded $4.6 Million NIH Grant For Chronic Pain Research

University of Illinois at Chicago awarded $4.6 million NIH grant for chronic pain research

California’s Stem Cell Agency Invests in Stem Cell-Based Therapies Targeting Sickle Cell Disease and Cancer

Sickle Cell Disease Association Of America, Inc. Awards Community Based Organizations With $2,033,080 For Newborn Screening Follow-Up Program

Sickle Cell Disease Association of America, Inc. Awards Community Based Organizations With $2,033,080 for Newborn Screening Follow-Up Program

Ex-rutgers Stars Devin And Jason McCourty Top $1M In Fundraising For Charity

Ex-rutgers stars Devin and Jason McCourty top $1M in fundraising for charity

Emmaus Medical, Inc. Selects AmerisourceBergen To Support The Launch Of EndariTM (L-glutamine Oral Powder)

Emmaus Medical, Inc. Selects AmerisourceBergen to Support the Launch of EndariTM (L-glutamine oral powder)

Bluebird Bio Acquires Durham, NC, Manufacturing Site For Lentiviral Vector Production

Bluebird bio acquires Durham, NC, manufacturing site for lentiviral vector production

Cydan Raises $34M To Advance New Therapies For Sickle Cell, Other Rare Diseases

Cydan Raises $34M to Advance New Therapies for Sickle Cell, Other Rare Diseases

FDA Awards UNC Researcher $2M Grant To Study Kidney Disease In Sickle Cell Anemia

FDA Awards UNC Researcher $2M Grant to Study Kidney Disease in Sickle Cell Anemia

Matthew Porteus Awarded Grant For Sickle Cell Anemia Work

Matthew Porteus awarded grant for sickle cell anemia work

DDCF Awards $6 Million For Sickle Cell Disease Research

DDCF Awards $6 Million for Sickle Cell Disease Research

PCORI Board Approves $18 Million For Research On Sickle Cell Disease

PCORI Board Approves $18 Million for Research on Sickle Cell Disease

Bioverativ And Bicycle Therapeutics Enter Into Strategic Research Collaboration To Develop Therapies For Hemophilia And Sickle Cell Disease

Bioverativ and Bicycle Therapeutics Enter into Strategic Research Collaboration to Develop Therapies for Hemophilia and Sickle Cell Disease

CSL Behring To Acquire Biotech Company Calimmune And Its Proprietary Stem Cell Gene Therapy Platform

CSL Behring to Acquire Biotech Company Calimmune and its Proprietary Stem Cell Gene Therapy Platform

Gamida Cell Announces $3.5 Million Grant From The Israeli Government

Gamida Cell Announces $3.5 Million Grant from the Israeli Government

Sickle Cell Disease Association Of America, Inc. Awarded $11.6 Million Continuation Grant From The Health Resources & Services Administration

Sickle Cell Disease Association of America, Inc. Awarded $11.6 Million Continuation Grant from the Health Resources & Services Administration

GlycoMimetics Announces Pricing Of Public Offering Of Common Stock

GlycoMimetics Announces Pricing of Public Offering of Common Stock

Apheresis Equipment Market Is Expected To Reach USD 3.7 Billion By 2024

Apheresis Equipment Market is expected to reach USD 3.7 billion by 2024

Global HOPE Initiative Plans $100M Pediatric Hematology-Oncology Treatment Network In Africa

Global HOPE Initiative Plans $100M Pediatric Hematology-Oncology Treatment Network in Africa

U.S. Government Awards Missouri Researcher $4.3 Million To Study Sickle Cell In Teens, Adults

U.S. Government Awards Missouri Researcher $4.3 Million to Study Sickle Cell in Teens, Adults

Exemplar Genetics Awarded Subcontract To Advance New Therapeutic Models For Sickle Cell Disease

Exemplar Genetics Awarded Subcontract to Advance New Therapeutic Models for Sickle Cell Disease

Calimmune Expands Lentiviral Gene Therapy Pipeline Through License Of Sickle Cell Disease Therapeutic Candidate

Calimmune Expands Lentiviral Gene Therapy Pipeline Through License of Sickle Cell Disease Therapeutic Candidate

Novartis Acquires US Pharma Research Firm Selexys Pharmaceuticals

Novartis acquires US pharma research firm Selexys Pharmaceuticals

183 resources available:

The CRISPR Era Is Here

The CRISPR Era Is Here

Vaso-Occlusive Crisis: The Future Of Treating Patients With Sickle Cell Disease In Emergency Departments

Vaso-Occlusive Crisis: The Future of Treating Patients With Sickle Cell Disease in Emergency Departments

Corinna Schultz, MD, Shares How Pediatricians Can Discuss Sickle Cell Trait With Patients, Families

Corinna Schultz, MD, Shares How Pediatricians Can Discuss Sickle Cell Trait With Patients, Families

From Early Hypothesis To Clinical Data About Reducing Vaso-Occlusive Episodes With Arginine Therapy

From Early Hypothesis to Clinical Data About Reducing Vaso-Occlusive Episodes with Arginine Therapy

Corinna Schultz, MD: Does Sickle Cell Trait Get Discussed?

Corinna Schultz, MD: Does Sickle Cell Trait Get Discussed?

Claudia Morris, MD, Uncovers Potential New Mechanism Of Action For Hydroxyurea

Claudia Morris, MD, Uncovers Potential New Mechanism of Action for Hydroxyurea

Arginine Therapy Shows Positive Impacts on Sickle Cell Disease in ASH 2022 Data

MARAC Statement On Influenza, National Shortage Of Oseltamivir (Tamiflu) And The Influenza Vaccine

MARAC Statement on Influenza, National Shortage of Oseltamivir (Tamiflu) and the Influenza Vaccine

Serious Gum Disease With Sickle Cell May Lead To Repeat Pain Crises

Serious Gum Disease With Sickle Cell May Lead to Repeat Pain Crises

Lab-Grown Blood: What Is It, And How Could It Be Useful?

Lab-Grown Blood: What Is It, and How Could It Be Useful?

MARAC Encourages Clinical Research Studies

MARAC Encourages Clinical Research Studies

Evaluation of Ovarian Reserve, Aging and Fertility Preservation in Women With Sickle Cell Disease

SCDAA News Advisory: Partial Hold On Gene Therapy Trial

SCDAA News Advisory: Partial Hold on Gene Therapy Trial

Gene Therapy May Help Cure Sickle Cell Disease, Study Says

Gene therapy may help cure sickle cell disease, study says

Experimental Gene Therapy Reverses Sickle Cell Disease For Years

Experimental Gene Therapy Reverses Sickle Cell Disease for Years

CTX001 Continues To Show Promise In Severe SCD

CTX001 Continues to Show Promise in Severe SCD

MARAC Advisory Statement: Gene Therapy & Bone Marrow Therapies

MARAC Advisory Statement: Gene Therapy & Bone Marrow Therapies

Statement On NHLBI Decision To Pause The Pilot And Feasibility Study Of Hematopoietic Stem Cell Gene Transfer For Sickle Cell Disease

Statement on NHLBI Decision to Pause the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease

FDA Clears Graphite Bio To Begin Trial For Gene Therapy In Sickle Cell Disease

FDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell Disease

SCD Patients Benefit From Early Rivipansel Treatment For VOCs, New Analyses Show

SCD Patients Benefit From Early Rivipansel Treatment for VOCs, New Analyses Show

Gene-Editing Treatment Shows Promise For Sickle Cell Disease

Gene-Editing Treatment Shows Promise for Sickle Cell Disease

Orphan Drug Designation Granted For CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy For Sickle Cell Disease

Orphan Drug Designation Granted for CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy for Sickle Cell Disease

Gut Microbiome Translates Stress Into Sickle Cell Crises

Gut Microbiome Translates Stress Into Sickle Cell Crises

Bluebird Bio Presents Data From LentiGlobin Gene Therapy Trial

Bluebird Bio Presents Data From LentiGlobin Gene Therapy Trial

Partnership To Ensure Supply Of LentiGlobin, Potential Gene Therapy

Partnership to Ensure Supply of LentiGlobin, Potential Gene Therapy

Expert Opinion Hydroxyurea Could Prevent Strokes For People With Sickle Cell Disease In Lieu Of Transfusions During COVID-19 Blood Supply Shortage

Expert Opinion Hydroxyurea Could Prevent Strokes for People With Sickle Cell Disease in Lieu of Transfusions During COVID-19 Blood Supply Shortage

At 16, She’s A Pioneer In The Fight To Cure Sickle Cell Disease

At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease

A Teenager’s Breakthrough Gene Therapy For Sickle Cell Disease

A Teenager’s Breakthrough Gene Therapy for Sickle Cell Disease

ASH and FDA Unveil New Recommendations to Guide Clinical Development of Sickle Cell Disease Therapies

Gene-Edited ‘Supercells’ Make Progress In Fight Against Sickle Cell Disease

Gene-Edited ‘Supercells’ Make Progress In Fight Against Sickle Cell Disease

FDA Awards $2M To Phase 2 Trial Of Vitamin D For Reducing Risk Of SCD Respiratory Complications

FDA Awards $2M to Phase 2 Trial of Vitamin D for Reducing Risk of SCD Respiratory Complications

Phase 3 Trial Of Rivipansel In Treating SCD Pain Crisis Fails To Meet Goals, Pfizer Announces

Phase 3 Trial of Rivipansel in Treating SCD Pain Crisis Fails to Meet Goals, Pfizer Announces

Sickle Cell Trait May Not Increase The Risk Of Death

Sickle cell trait may not increase the risk of death

Sickle Cell Patient Receives CRISPR Gene Therapy

Sickle Cell Patient Receives CRISPR Gene Therapy

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

Non-Invasive Prenatal Screening Test For Sickle Cell Appears Possible, Study Reports

Non-Invasive Prenatal Screening Test for Sickle Cell Appears Possible, Study Reports

UH Researcher Reports The Way Sickle Cells Form May Be Key To Stopping Them

UH Researcher Reports the Way Sickle Cells Form May Be Key to Stopping Them

Investigational Oral Inhibitor IMR-687 Shows Promising Results In Ongoing Phase 2 Clinical Trial

Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical Trial

Cognitive Function May Be Affected In Adults With Sickle Cell Disease, Study Suggests

Cognitive Function may be Affected in Adults With Sickle Cell Disease, Study Suggests

More Online Queries In Winter Suggest Seasonal Variations In SCD Activity

More Online Queries in Winter Suggest Seasonal Variations in SCD Activity

More Curative Bone Marrow Transplants Are Successful When Patients Given Double The Radiation Beforehand, Study Says

More Curative Bone Marrow Transplants are Successful When Patients Given Double the Radiation Beforehand, Study Says

Johns Hopkins Researchers Offer New Protocol To Potentially Cure Sickle Cell Disease

Johns Hopkins researchers offer new protocol to potentially cure sickle cell disease

Big Jump in Success for Sickle Cell Transplants

Could Gene Therapy Cure Sickle Cell Anemia?

Could Gene Therapy Cure Sickle Cell Anemia?

Differences In Brain Oxygen Supply May Explain Silent Strokes In SCD Patients

Differences in Brain Oxygen Supply May Explain Silent Strokes in SCD Patients

Patients With Sickle Cell Disease May Have Lower Risk For C. Difficile

Patients With Sickle Cell Disease may Have Lower Risk for C. Difficile

Spectra Optia Device Save Way To Treat Sickle Cell While Using Less Blood Cell Packs, Study Finds

Spectra Optia Device Save Way to Treat Sickle Cell While Using Less Blood Cell Packs, Study Finds

Kids With Sickle Cell Anemia Are More Sedentary Than Healthy Peers, Study Suggests

Kids With Sickle Cell Anemia are More Sedentary Than Healthy Peers, Study Suggests

Virtual Reality Helps Reduce Pain Among Patients With Sickle Cell Disease

Virtual Reality Helps Reduce Pain Among Patients With Sickle Cell Disease

These Patients Had Sickle-cell Disease. Experimental Therapies Might Have Cured Them

These patients had sickle-cell disease. Experimental therapies might have cured them

Vaso-Occlusive Pain Linked To Menstruation In Some Patients, Study Finds

Vaso-Occlusive Pain Linked to Menstruation in Some Patients, Study Finds

Safety And Early Hints Of Benefit Seen In Phase 1b Trial Of PF-04447943

Safety and early hints of benefit seen in phase 1b trial of PF-04447943

Randomized Trials Needed To Assess Benefits Of Salmonella Vaccines In SCD Patients, Study Says

Randomized Trials Needed to Assess Benefits of Salmonella Vaccines in SCD Patients, Study Says

Toronto Neuroscientist Getting Closer To Tailored Treatments For Chronic Pain

Toronto Neuroscientist Getting Closer to Tailored Treatments for Chronic Pain

Crizanlizumab Designated FDA Breakthrough Therapy For Potential In Vaso-occlusive Crisis Prevention

Crizanlizumab designated FDA breakthrough therapy for potential in vaso-occlusive crisis prevention

CTX001 Earns FDA’s Fast Track Status For Treating Sickle Cell Disease

CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell Disease

FDA Grants CRISPR Gene Therapy Fast Track Designation For Sickle Cell Disease

FDA grants CRISPR gene therapy fast track designation for sickle cell disease

Hospitals See No Link Between US Opioid Crisis And Patients’ Use Of Treatment, Study Reports

Hospitals See No Link Between US Opioid Crisis and Patients’ Use of Treatment, Study Reports

ASH Launches Sickle Cell Disease Clinical Trials Network To Accelerate Therapy Development

ASH launches sickle cell disease clinical trials network to accelerate therapy development

Boston Children’s Hospital Receives Grant For Sickle Cell Disease Research

Boston Children’s Hospital receives grant for sickle cell disease research

LentiGlobin Shows Positive Effects In Severe Sickle Cell Disease Patients, Phase 1/2 Data Reports

LentiGlobin Shows Positive Effects in Severe Sickle Cell Disease Patients, Phase 1/2 Data Reports

Gene Therapy Targets Sickle-cell Disease

Gene therapy targets sickle-cell disease

Voxelotor Can Promote Long-Term Benefits In Teens, Adults With SCD, Latest Clinical Data Show

Voxelotor can Promote Long-Term Benefits in Teens, Adults With SCD, Latest Clinical Data Show

Study Confirms Safe Use of Opioids for Pain Control in Sickle Cell Disease

Inexpensive Sickle Cell Diagnostic Tool Shows Perfect Accuracy In Uganda

Inexpensive sickle cell diagnostic tool shows perfect accuracy in Uganda

Early Clinical Trial Data Show Gene Therapy Reversing Sickle Cell Anemia

Early clinical trial data show gene therapy reversing sickle cell anemia

Gene Therapies Could Transform The Treatment Of Sickle Cell Disease

Gene therapies could transform the treatment of sickle cell disease

School Staff Lacks Knowledge About Sickle Cell Disease, Student Survey Finds

School staff lacks knowledge about sickle cell disease, student survey finds

Phase 1 Trial To Test Under-the-skin Injection Of Sevuparin In Sickle Cell Patients

Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patients

Research Team Recognized For Organ-on-a-Chip Design

Research Team Recognized for Organ-on-a-Chip Design

Marijuana Use Common In Sickle Cell Patients, Highlighting Need For More Research, Study Shows

Marijuana use common in sickle cell patients, highlighting need for more research, study shows

Crizanlizumab Lowers Pain Crises In At-risk Sickle Cell Patients, Ad-hoc Trial Data Show

Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data show

Sickle Cell Patients In UK Survey, Especially Those 16 To 20, Voice Problems With Care And Pain Relief Given

Sickle cell patients in UK survey, especially those 16 to 20, voice problems with care and pain relief given

American Society Of Hematology To Launch Sickle Cell Disease Clinical Trials Network

American Society of Hematology to launch sickle cell disease clinical trials network

How Sickled Red Blood Cells Stick To Blood Vessels

How sickled red blood cells stick to blood vessels

Grant Recipient Uses Mobile Device Observation In Sickle Cell Pain Study

Grant Recipient Uses Mobile Device Observation in Sickle Cell Pain Study

Sickle Cell Treatments Can Destroy Germ Cells In Boys, Affecting Fertility In Adulthood, Study Suggests

Sickle Cell Treatments can Destroy Germ Cells in Boys, Affecting Fertility in Adulthood, Study Suggests

Mount Sinai Receives NIH Grant To Study Use Of Inhaled Corticosteroids For Sickle Cell Treatment

Mount Sinai receives NIH grant to study use of inhaled corticosteroids for sickle cell treatment

L-glutamine Oral Powder Significantly Reduces Acute Complications Of Sickle Cell Disease

L-glutamine Oral Powder Significantly Reduces Acute Complications of Sickle Cell Disease

Endari Reduces Pain Crises, Hospitalizations In Sickle Cell Patients, Phase 3 Trial Shows

Endari reduces pain crises, hospitalizations in sickle cell patients, phase 3 trial shows

Researchers Raise Funds For Phase 1 Trial To Test Medical Cannabis In Sickle Cell Disease

Researchers raise funds for phase 1 trial to test medical cannabis in sickle cell disease

Music Therapy Helps Relieve Pain In Adults With Sickle Cell Disease, Pilot Trial Suggests

Music therapy helps relieve pain in adults with sickle cell disease, pilot trial suggests

GBT Announces Positive Top-line Data From Part A Of The Phase 3 HOPE Study Of Voxelotor In Sickle Cell Disease

GBT announces positive top-line data from part A of the phase 3 HOPE study of Voxelotor in sickle cell disease

Potential Treatment Targeting Nitric Oxide Levels, Olinciguat, Named Orphan Drug By FDA

Potential treatment targeting nitric oxide levels, olinciguat, named orphan drug by FDA

Global Blood Therapeutics (GBT) Announces Upcoming Data Presentations Supporting Voxelotor SCD Program

Global Blood Therapeutics (GBT) announces upcoming data presentations supporting Voxelotor SCD program

Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation For Olinciguat For The Treatment Of Sickle Cell Disease

Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disease

Gene-Therapy Company Crispr Drops As FDA Puts Trial On Hold

Gene-Therapy Company Crispr Drops as FDA Puts Trial on Hold

Kids With Sickle Cell Disease Aren’t Receiving Key Vaccines, Michigan Study Finds

Kids with sickle cell disease aren’t receiving key vaccines, Michigan study finds

Stigmatizing Language In Medical Records Affects Patient Care, Study Shows

Stigmatizing language in medical records affects patient care, study shows

Sickle Cell Groups, Pfizer Work To Bring African-Americans Into Clinical Trials

Sickle Cell Groups, Pfizer Work to Bring African-Americans into Clinical Trials

Sickle Cell Trait May Not Contribute To Stroke Risk

Sickle cell trait may not contribute to stroke risk

Is Caregiver Education About Sickle Cell Trait Effective?

Is Caregiver Education About Sickle Cell Trait Effective?

Healthy Red Blood Cells Owe Their Shape To Muscle-like Structures

Healthy red blood cells owe their shape to muscle-like structures

Solution To 50-year-old Mystery Could Lead To Gene Therapy For Common Blood Disorders

Solution to 50-year-old mystery could lead to gene therapy for common blood disorders

Sancilio Pharmaceuticals Company Announces Altemia™ Receives Orphan Drug Designation From The European Medicines Agency For The Treatment Of Pediatric Patients With Sickle Cell Disease (SCD)

Sancilio Pharmaceuticals Company announces Altemia™ receives orphan drug designation from the European Medicines Agency for the treatment of pediatric patients with Sickle Cell Disease (SCD)

FDA Approves Hydroxyurea Tablets For Pediatric Patients With Sickle Cell Anemia

FDA Approves Hydroxyurea Tablets for Pediatric Patients With Sickle Cell Anemia

ADDMEDICA Receives FDA-approval For Orphan Drug Siklos®, First And Sole Hydroxyurea-based Treatment For Paediatric Patients With Sickle Cell Anaemia

ADDMEDICA receives FDA-approval for orphan drug Siklos®, first and sole hydroxyurea-based treatment for paediatric patients with sickle cell anaemia

How One Child’s Sickle Cell Mutation Helped Protect The World From Malaria

How One Child’s Sickle Cell Mutation Helped Protect the World From Malaria

Clinical Trial Offers Hope To Adults With Sickle Cell Disease

Clinical trial offers hope to adults with sickle cell disease

The Impact Of Rare Disease On Family Caregivers

The impact of rare disease on family caregivers

Gamida Cell To Present Data From NiCord® Programs At The 2018 BMT Tandem Meetings

Gamida Cell to Present Data from NiCord® Programs at the 2018 BMT Tandem Meetings

Kids Who Need Sickle Cell Meds Don’t Always Get Them

Kids Who Need Sickle Cell Meds Don’t Always Get Them

Progress In Pursuit Of Sickle Cell Cure

Progress in pursuit of sickle cell cure

First Sickle Cell Patient Dosed In Phase 2a Of IMR-687

First Sickle Cell Patient Dosed in Phase 2a of IMR-687

Hypersensitivity To Allergens May Increase Risk Of Acute Chest Syndrome In Sickle Cell Anemia Children

Hypersensitivity to Allergens May Increase Risk of Acute Chest Syndrome in Sickle Cell Anemia Children

University Researchers Develop More Complete Model Of Sickle Cell

University researchers develop more complete model of sickle cell

Hydroxyurea Treatment In Men With SCA Leads To Drop In Total Sperm Count, Study Shows

Hydroxyurea Treatment in Men with SCA Leads to Drop in Total Sperm Count, Study Shows

Sickle Cell Treatment Endari Now Available In The United States

Sickle Cell Treatment Endari Now Available in the United States

GBT Receives FDA Breakthrough Therapy Designation For Voxelotor For Treatment Of Sickle Cell Disease (SCD)

GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)

La Jolla Pharmaceutical Company Announces Initiation Of Pivotal Clinical Study Of LJPC-401 In Patients With Beta Thalassemia

La Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta thalassemia

Emmaus Life Sciences, Inc. To Present Results Of Phase 3 Study Of EndariTM (L-glutamine Oral Powder) At 59th American Society Of Hematology Annual Meeting

Emmaus Life Sciences, Inc. to present results of phase 3 study of endariTM (L-glutamine oral powder) at 59th American Society of Hematology annual meeting

Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is Safe

Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is Safe

Sickle Cell Patient With Severe Anemia Rapidly Improves With Voxelotor, Case Study Shows

Sickle cell patient with severe anemia rapidly improves with voxelotor, case study shows

Global Blood Therapeutics Announces Completion Of Safety Review By Independent Data And Safety Monitoring Board (DSMB) For Phase 3 HOPE Study In Sickle Cell Disease

Global Blood Therapeutics Announces Completion of Safety Review by Independent Data and Safety Monitoring Board (DSMB) for Phase 3 HOPE Study in Sickle Cell Disease

Alzheimer’s Treatment Memantine Shows Promise In Treating Sickle Cell Disease

Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell Disease

Investigational Therapy Altemia Achieves Main Endpoints In Pediatric Sickle Cell Trial

Investigational Therapy Altemia Achieves Main Endpoints in Pediatric Sickle Cell Trial

Georgia Universities Join NIH-funded National Study Of Bone Marrow Transplant For SCD

Georgia Universities Join NIH-funded National Study of Bone Marrow Transplant for SCD

Hydroxyurea Linked To ‘significant, Rapid’ Reduction Of Sperm Count

Hydroxyurea linked to ‘significant, rapid’ reduction of sperm count

SCA Therapy Hydroxyurea Doesn’t Boost Malaria Risk In Sub-Saharan Africa, Study Finds

SCA Therapy Hydroxyurea Doesn’t Boost Malaria Risk in Sub-Saharan Africa, Study Finds

Fighting Sickle Cell Disease By Looking Back To Babyhood

Fighting sickle cell disease by looking back to babyhood

National Sickle Cell Disease Poll Of African Americans Dispels Long-Held Views

National Sickle Cell Disease Poll of African Americans Dispels Long-Held Views

Sancilio Pharmaceuticals Company, Inc. (SPCI) Receives Rare Pediatric Disease Designation From The US Food And Drug Administration For Altemia, A Treatment Of Sickle Cell Disease (SCD) In Children

Sancilio Pharmaceuticals Company, Inc. (SPCI) Receives Rare Pediatric Disease Designation From the US Food and Drug Administration for Altemia, a Treatment of Sickle Cell Disease (SCD) in Children

Imara Reports Favorable Preclinical And Phase 1 Data On IMR-687 In Sickle Cell Disease

Imara Reports Favorable Preclinical and Phase 1 Data on IMR-687 in Sickle Cell Disease

Computer Models Provide New Understanding Of Sickle Cell Disease

Computer models provide new understanding of sickle cell disease

‘Natural’ Gene Mutation May Offer Way Of Treating Sickle Cell Disease, Study Says

‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study Says

Sancilio Pharmaceuticals Company, Inc. Announces Completion Of Enrollment In The SCOT Trial In Pediatric Patients With Sickle Cell Disease Using Altemia Capsules

Sancilio Pharmaceuticals Company, Inc. Announces Completion of Enrollment in the SCOT Trial in Pediatric Patients With Sickle Cell Disease Using Altemia Capsules

Quest Diagnostics Launches Genetic Test To Assess Risk For Sickle Cell Anemia, Other Inherited Disorders

Quest Diagnostics Launches Genetic Test to Assess Risk for Sickle Cell Anemia, Other Inherited Disorders

FDA Approves Glutamine Powder for Sickle Cell Disease

Researchers Identify Genetic Predictors Of Sickle Cell Anemia-Related Complications

Researchers Identify Genetic Predictors of Sickle Cell Anemia-Related Complications

Global Blood Therapeutics Receives EMA PRIME Designation For GBT440 For The Treatment Of Sickle Cell Disease (SCD)

Global Blood Therapeutics Receives EMA PRIME Designation for GBT440 for the Treatment of Sickle Cell Disease (SCD)

Chip-based Models Mimic Organs To Advance Understanding Of Sickle Cell Disease

Chip-based Models Mimic Organs to Advance Understanding of Sickle Cell Disease

Small Chips, Big Impact: MSU Researcher Studies Cardiovascular, Sickle Cell Disease

Small chips, big impact: MSU researcher studies cardiovascular, sickle cell disease

ASH To Develop Clinical Guidelines To Improve Care For People With Sickle Cell Disease

ASH to develop clinical guidelines to improve care for people with sickle cell disease

Only 21% Of Kids With ADHD And SCD Are Treated For Attention Deficit

Only 21% of Kids with ADHD and SCD Are Treated for Attention Deficit

New Pre-transplant Treatment Regimen Improves Survival Of Kids With Sickle Cell Disease, Trial Shows

New Pre-transplant Treatment Regimen Improves Survival of Kids with Sickle Cell Disease, Trial Shows

Researchers Develop Test For SCA To Be Used In Low-Resource Settings

Researchers Develop Test for SCA to be Used in Low-Resource Settings

Multidisciplinary Care Team Greatly Reduces Risk In Pregnant SCD Patients In Low-income Countries

Multidisciplinary Care Team Greatly Reduces Risk in Pregnant SCD Patients in Low-income Countries

FDA Advisory Committee Recommends Approval Of Endari™ From Emmaus Life Sciences For The Treatment Of Sickle Cell Disease

FDA Advisory Committee Recommends Approval of Endari™ from Emmaus Life Sciences for the Treatment of Sickle Cell Disease

New Method Of Creating Healthy Stem Cells Could Potentially Improve Treatment Of Sickle Cell Anemia

New Method of Creating Healthy Stem Cells Could Potentially Improve Treatment of Sickle Cell Anemia

Imara’s IMR-687 For SCD Receives FDA Rare Pediatric Disease Designation

Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease Designation

Phase 3 Trial Recruiting To Test Rivipansel For Vaso-Occlusive Crisis In SCD

Phase 3 Trial Recruiting to Test Rivipansel for Vaso-Occlusive Crisis in SCD

Sickle Cell Disease And Its Toll Compared In Different Age Groups In Study

Sickle Cell Disease and Its Toll Compared in Different Age Groups in Study

Prolong Pharma’s Sanguinate Shows Promise In Reverting Shape Of Red Blood Cells In SCD

Prolong Pharma’s Sanguinate Shows Promise in Reverting Shape of Red Blood Cells in SCD

New Research Suggests SANGUINATE™ Reduces The Number Of Sickled Red Blood Cells In Patients With Vaso-Occlusive Crisis

New Research Suggests SANGUINATE™ Reduces the Number of Sickled Red Blood Cells in Patients with Vaso-Occlusive Crisis

MaxCyte, Inc. To Present Positive Preclinical Data For Sickle Cell Disease

MaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell Disease

First systemic evidence for safety of tPA in stroke patients with sickle cell disease

Young Sickle Cell Patients Who Don’t Take Medication Have Lower Quality Of Life

Young Sickle Cell Patients Who Don’t Take Medication Have Lower Quality of Life

Older Blood Used In Transfusions May Be Harmful To Adult Sickle Cell Patients, Review Finds

Older Blood Used in Transfusions May Be Harmful to Adult Sickle Cell Patients, Review Finds

Sickle cell gene linked to elevated risk of developing kidney failure

In France, Boy Becomes First Sickle Cell Disease Patient To Be Treated With Gene Therapy

In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene Therapy

Sickle Cell Anemia Toddlers Benefit From Maximal Hydroxyurea Doses, Study Finds

Sickle Cell Anemia Toddlers Benefit from Maximal Hydroxyurea Doses, Study Finds

Bluebird Bio Announces Publication Of Case Study On First Patient With Severe Sickle Cell Disease Treated With Gene Therapy In The New England Journal Of Medicine

bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene Therapy in The New England Journal of Medicine

Study Suggests Ways Of Improving Newborn Screening For Sickle Cell Disease

Study Suggests Ways of Improving Newborn Screening for Sickle Cell Disease

Sickle Cell Trait May Confound Blood Sugar Readings Among African-Americans

Sickle cell trait may confound blood sugar readings among African-Americans

A Multiple Drug Approach To Preventing Sickle Cell Crisis

A multiple drug approach to preventing sickle cell crisis

World’s Largest Sickle Cell Disease Stem Cell Library Underway

World’s Largest Sickle Cell Disease Stem Cell Library Underway

Diabetes Drug, Metformin, Suggested As ‘Breakthrough’ Treatment For Sickle Cell Anemia

Diabetes Drug, Metformin, Suggested as ‘Breakthrough’ Treatment for Sickle Cell Anemia

SPCI Launched The SCOT (Sickle Cell Omega-3 Treatment) Trial In The United States With An Investigator Meeting Held December 16-17 In Atlanta

SPCI Launched the SCOT (Sickle Cell Omega-3 Treatment) Trial in the United States with an Investigator Meeting Held December 16-17 in Atlanta

SUSTAIN Clinical Trial Results Show Crizanlizumab Reduced Sickle Cell–Related Pain Crises

SUSTAIN Clinical Trial Results Show Crizanlizumab Reduced Sickle Cell–Related Pain Crises

Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia

Sickle Cell Disease Research Shows Progress In Preventing Related Complications And Death

Sickle Cell Disease Research Shows Progress in Preventing Related Complications and Death

Bluebird Bio Presents New Data From HGB-205 Study Of LentiGlobinTM Drug Product In Patients With Transfusion-Dependent β-Thalassemia (TDT) And Severe Sickle Cell Disease At American Society Of Hematology (ASH) Annual Meeting

bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting

Preclinical Data On Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness In The Treatment Of Sickle Cell Disease Presented At The American Society Of Hematology Annual Meeting

Preclinical Data on Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness in the Treatment of Sickle Cell Disease Presented at the American Society of Hematology Annual Meeting

Step Toward Gene Therapy For Sickle Cell Disease

Step toward gene therapy for sickle cell disease

Stem Cell Gene-editing Method May Be Breakthrough For Sickle Cell Research

Stem cell gene-editing method may be breakthrough for sickle cell research

Environmental, Genetic Factors May Predict Longevity In Sickle Cell Disease

Environmental, genetic factors may predict longevity in sickle cell disease

CRISPR Deployed To Combat Sickle-cell Anaemia

CRISPR deployed to combat sickle-cell anaemia

Methadone Provides Pain Relief For Kids With Sickle Cell

Methadone provides pain relief for kids with sickle cell

Rare Patients With Sickle Cell Disease Live Nearly Twice National Average

Rare patients with sickle cell disease live nearly twice national average

Effort Set To Help Sickle Cell Patients Manage Meds

Effort set to help sickle cell patients manage meds

BCL11A-based Gene Therapy For Sickle Cell Disease Passes Key Preclinical Test

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

Researchers ID key drivers of heart complications in sickle cell anemia