Updates

July 16, 2019July 16, 2019
‘Sickle Cell Speaks’ Campaign Raises Awareness with Aim of Eroding Stigmas, GBT and Partners Say

In partnership with community organizations, Global Blood Therapeutics (GBT) is directing a national campaign to raising awareness and understanding of sickle cell disease, ... Read more
July 11, 2019July 11, 2019
Non-Invasive Prenatal Screening Test for Sickle Cell Appears Possible, Study Reports

A non-invasive prenatal test to diagnose a risk of sickle cell disease in a baby is possible and may be available in clinics in ... Read more
July 9, 2019July 9, 2019
45th Annual SCDAI 8K Walk, Jog, or Bike-a-Thon Set for July 20 in Chicago

The Sickle Cell Disease Association of Illinois (SCDAI) announced plans to hold its 45th Annual 8K Walk/Jog/Bike-A-Thon on July 20, continuing the group’s ... Read more
July 4, 2019
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more
July 2, 2019July 2, 2019
Chronic Pain in Sickle Cell Disease Underpinned by Changes in Brain Networks, Study Suggests

Chronic pain in adults with sickle cell disease (SCD), a frequent symptom of the disease, is associated with alterations in the connection of brain ... Read more

News

Early Treatment with Hydroxyurea Lessened SCD Severity Among Infants, Study Shows
June 27, 2019June 27, 2019
Treatment with hydroxyurea since early infancy, starting at the age of one, is effective ...
News

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
June 26, 2019
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ...
News

Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical Trial
June 25, 2019June 25, 2019
Imara’s investigational oral inhibitor IMR-687 is well-tolerated, and shows signs of potential for lowering ...
News

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 20, 2019
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ...
News

HOPE Trial Shows Voxelotor May Increase Hemoglobin, Reduce Anemia in Adults and Adolescents With SCD
June 20, 2019June 20, 2019
Treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can markedly reduce both hemolysis — the ...
News

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
June 18, 2019
People with rare diseases know that the right government policies can make a big ...

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Howard University Symposium Focuses on Sickle Cell Cure
June 18, 2019June 18, 2019
Howard University’s 6th Annual World Sickle Cell Awareness Day Symposium has its sights set on ...
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 ...
GBT Awards $200K in ACCEL Grants to Improve SCD Healthcare Delivery
June 11, 2019June 11, 2019
Sickle Cell Advocate Howard Woolley Wins Prestigious Award for Working to Improve Patient Care
June 6, 2019June 6, 2019
SCDAA Plans June 22 ‘Walk With the Stars’ Fundraiser in Baltimore
June 4, 2019June 4, 2019
FDA Grants Fast Track Status to Imara’s IMR-687 Candidate Therapy for Sickle Cell Disease
May 30, 2019May 30, 2019

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 ...
Novartis’ STEP Program Funding Projects Supporting Sickle Cell Community
May 28, 2019May 28, 2019
Participants Sought for Trial Evaluating Olinciguat for Sickle Cell Disease
May 23, 2019May 23, 2019
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
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
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

News

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 ...
News

Findings on Abnormal Hemoglobin Assembly Could Lead to Lower-Dose SCD Medications, Researchers Say
May 2, 2019
A study of sickle cell disease (SCD) at the molecular level found abnormal hemoglobin ...
News

Holistic Treatment Approach May Help Heal Long-Lasting SCD-Related Skin Ulcers, Case Report Shows
April 30, 2019April 30, 2019
A newly developed treatment approach, known by the acronym SICKLE, was shown to be ...
News

More Online Queries in Winter Suggest Seasonal Variations in SCD Activity
April 25, 2019
More people search for information on sickle cell disease (SCD) during winter than summer, indicating ...
News

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 ...
News

NFL Broadcaster Huddles With Emmaus to ‘Sideline Sickle Cell’ Disease
April 23, 2019April 23, 2019
In an effort to raise awareness about the latest in sickle cell disease therapy, ...
News

Loma Linda Hospital ‘Effecively Cures’ Girl Using Stem Cells Transplanted from Dad, Report Says
April 18, 2019April 18, 2019
Clinicians at the Loma Linda University Children’s Hospital successfully performed that institute’s first stem cell transplant, and ...
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 ...

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