News

August 22, 2019August 22, 2019

ReachBio Launches Screening Platform to Find New Therapies for SCD

News

ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that trigger the production of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and other ... Read more

August 20, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 20, 2019August 22, 2019

ICER Will Compare Lead Candidate Therapies Crizanlizumab and Voxelotor for Efficacy, Value

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An upcoming analysis by the Institute for Clinical and Economic Review (ICER) will compare the clinical efficacy and economic value of Novartis’ crizanlizumab and Global Blood Therapeutics‘ voxelotor, lead ... Read more

August 15, 2019August 15, 2019

New Technology May Allow for Better Monitoring of Sickle Cell Disease

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A new way to monitor blood cells that doesn’t require microscopic imaging or biochemical markers may allow for better management of sickle cell disease, a study suggests. The study, ... Read more

August 13, 2019August 13, 2019

Cognitive Decline Not Evident in Black Americans with Sickle Cell Trait, Study Finds

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African-Americans with sickle cell trait, carriers for sickle cell but without the disease, have no greater risk of cognitive decline than other adults of similar age, an analysis of a ... Read more

August 8, 2019August 8, 2019

Survival After Kidney Transplant Lower in SCD Patients Than Other Kidney Disease Patients, Analysis Shows

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Survival of sickle cell disease patients (SCD) with end-stage renal disease who undergo kidney transplant is significantly lower compared to other renal disease patients who have the same procedure, ... Read more

August 6, 2019August 6, 2019

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

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The Phase 3 RESET trial testing the effectiveness and safety of rivipansel (GMI-1070), Pfizer‘s investigational candidate to treat vaso-occlusive crisis (VOC) in hospitalized patients with sickle cell disease (SCD), failed to reach both ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

August 1, 2019August 1, 2019

NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and Trials

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Wide interest in a CBS 60 Minutes story about a Florida woman with sickle cell disease (SCD) whose symptoms were eased through gene therapy has prompted the National Heart, ... Read more