Updates

June 25, 2019June 25, 2019
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical Trial

Imara’s investigational oral inhibitor IMR-687 is well-tolerated, and shows signs of potential for lowering blood biomarkers of sickle cell anemia (SCA), early data ... Read more
June 20, 2019
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their ... Read more
June 20, 2019June 20, 2019
HOPE Trial Shows Voxelotor May Increase Hemoglobin, Reduce Anemia in Adults and Adolescents With SCD

Treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can markedly reduce both hemolysis — the destruction of red blood cells — and anemia in adolescents ... Read more
June 18, 2019
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those ... Read more
June 18, 2019June 18, 2019
Howard University Symposium Focuses on Sickle Cell Cure

Howard University’s 6th Annual World Sickle Cell Awareness Day Symposium has its sights set on a cure for the inherited blood disorder. To be held ... Read more

News

Sevuparin Fails to Show Clinically Meaningful Improvements in Managing VOCs, Phase 2 Study Shows
June 13, 2019June 13, 2019
Sevuparin, Modus Therapeutics‘ lead therapeutic candidate for managing painful vaso-occlusive crises (VOCs), failed to show ...
News

GBT Awards $200K in ACCEL Grants to Improve SCD Healthcare Delivery
June 11, 2019June 11, 2019
Global Blood Therapeutics (GBT) announced the five organizations that will receive more than $200,000 ...
News

Sickle Cell Advocate Howard Woolley Wins Prestigious Award for Working to Improve Patient Care
June 6, 2019June 6, 2019
Prominent sickle cell advocate and philathropist Howard Woolley has won a Sickle Cell Outstanding ...
News

SCDAA Plans June 22 ‘Walk With the Stars’ Fundraiser in Baltimore
June 4, 2019June 4, 2019
Robert and Tanique Mitchell still remember the moment they found out that their granddaughter, ...
News

FDA Grants Fast Track Status to Imara’s IMR-687 Candidate Therapy for Sickle Cell Disease
May 30, 2019May 30, 2019
IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation ...
News

Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
May 28, 2019May 28, 2019
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims ...

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Novartis’ STEP Program Funding Projects Supporting Sickle Cell Community
May 28, 2019May 28, 2019
Novartis‘ Solutions to Empower Patients (STEP) program will fund five nonprofit organizations that address ...
Participants Sought for Trial Evaluating Olinciguat for Sickle Cell Disease
May 23, 2019May 23, 2019
The Phase 2 STRONG-SCD clinical trial evaluating olinciguat as an investigational therapy for sickle ...
Stakeholders Call for Improved Care for SCD Patients in California
May 21, 2019May 21, 2019
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 16, 2019May 16, 2019
SCD Experts Call for Routine Screening Among Migrants
May 16, 2019May 16, 2019
Cognitive Function May Be Affected in Adults with Sickle Cell Disease, Study Suggests
May 14, 2019May 14, 2019

CIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell Disease
May 7, 2019May 7, 2019
The California Institute for Regenerative Medicine (CIRM) and the National Heart, Lung and Blood ...
Findings on Abnormal Hemoglobin Assembly Could Lead to Lower-Dose SCD Medications, Researchers Say
May 2, 2019
Holistic Treatment Approach May Help Heal Long-Lasting SCD-Related Skin Ulcers, Case Report Shows
April 30, 2019April 30, 2019
More Online Queries in Winter Suggest Seasonal Variations in SCD Activity
April 25, 2019

Nonprofit Group Works to Raise Rare Disease Awareness in India
April 25, 2019April 25, 2019
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years ...
NFL Broadcaster Huddles With Emmaus to ‘Sideline Sickle Cell’ Disease
April 23, 2019April 23, 2019
Loma Linda Hospital ‘Effecively Cures’ Girl Using Stem Cells Transplanted from Dad, Report Says
April 18, 2019April 18, 2019

News

Low Oxygen Regions in Brains of Sickle Cell Patients are Prone to Small Strokes, Study Shows
April 16, 2019April 16, 2019
Sickle cell disease patients experience silent strokes in the deep white matter of the ...
News

Abnormal Red Blood Cells’ Shape Affect Blood Clot Formation, Study Shows
April 11, 2019April 11, 2019
Abnormal sickle-shaped red blood cells characteristic of sickle cell disease (SCD) change the structure ...
News

Identifying Outcomes for Sickle Cell Disease Clinical Trials is Aim of coreSCD
April 9, 2019April 9, 2019
The Green Park Collaborative (GPC) is working with the Sickle Cell Disease Association of America (SCDAA) to ...
News

Pfizer Executive Outlines Vision for Treating Sickle Cell Disease, Other Rare Diseases
April 4, 2019April 4, 2019
Consumers may know Pfizer as the company that manufactures Viagra, but the New York-based pharma ...
News

Study Determines Low Adherence to Hydroxyurea Treatment Among Children, Adolescents
April 2, 2019April 2, 2019
Children and adolescents with sickle cell anemia (SCA) showed low adherence to treatment with ...
News

Alterations in Blood Vessels of the Lungs Should be Monitored in SCD Patients, Study Says
March 28, 2019March 28, 2019
Alterations in the lungs’ network of blood vessels, caused by sickle cell disease (SCD), ...
News

Problem-Solving Intervention May Help SCD Teens to Transition to Adulthood, Study Shows
March 26, 2019March 26, 2019
Development of problem-solving skills may help adolescents with sickle cell disease prepare for the ...
News

More Curative Bone Marrow Transplants are Successful When Patients Given Double the Radiation Beforehand, Study Says
March 21, 2019March 21, 2019
The success rate of bone marrow transplants for patients with sickle cell disease or ...
News

Oral Morphine is a Viable Treatment Option for SCD Patients Experiencing VOCs, Study Says
March 19, 2019March 19, 2019
Oral morphine treatment is a valid therapy option for patients with sickle cell disease ...

October 25, 2016October 25, 2016

Kids with Sickle Cell Disease at Risk for Lead Poisoning, NYC Study Shows

News

National and local policies have led to large reductions in lead poisoning cases overall in the U.S., but severe cases are still common among children. Recently, researchers at the New York City Department of Health and Mental Hygiene (DOHMH) investigated sources of exposure, risk factors, and ... Read more