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June 25, 2019June 25, 2019

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

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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 from a Phase 2 trial show. ... Read more

June 20, 2019

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

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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 caregivers learn about their conditions, ... Read more

June 20, 2019June 20, 2019

HOPE Trial Shows Voxelotor May Increase Hemoglobin, Reduce Anemia in Adults and Adolescents With SCD

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Treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can markedly reduce both hemolysis — the destruction of red blood cells — and anemia in adolescents and adults with sickle cell ... Read more

June 18, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most ... Read more

June 18, 2019June 18, 2019

Howard University Symposium Focuses on Sickle Cell Cure

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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 June 19 — the 10th ... Read more

June 13, 2019June 13, 2019

Sevuparin Fails to Show Clinically Meaningful Improvements in Managing VOCs, Phase 2 Study Shows

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Sevuparin, Modus Therapeutics‘ lead therapeutic candidate for managing painful vaso-occlusive crises (VOCs), failed to show clinically meaningful improvements in patients with sickle cell disease (SCD), Phase 2 clinical trial results ... Read more

June 11, 2019June 11, 2019

GBT Awards $200K in ACCEL Grants to Improve SCD Healthcare Delivery

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Global Blood Therapeutics (GBT) announced the five organizations that will receive more than $200,000 worth of grants through its new Access to Excellent Care for Sickle Cell Patients Pilot ... Read more

June 6, 2019June 6, 2019

Sickle Cell Advocate Howard Woolley Wins Prestigious Award for Working to Improve Patient Care

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Prominent sickle cell advocate and philathropist Howard Woolley has won a Sickle Cell Outstanding Service Award, presented recently at the 2019 Roland B. Scott Memorial Symposium. Themed “Preparing the ... Read more

June 4, 2019June 4, 2019

SCDAA Plans June 22 ‘Walk With the Stars’ Fundraiser in Baltimore

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Robert and Tanique Mitchell still remember the moment they found out that their granddaughter, Kiarra Roseburgh, would be born with sickle cell disease (SCD). “We realized that we needed ... Read more

May 30, 2019May 30, 2019

FDA Grants Fast Track Status to Imara’s IMR-687 Candidate Therapy for Sickle Cell Disease

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IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). It’s currently being evaluated in a Phase 2a ... Read more