The American Physiological Society (APS) will host the Physiological and Pathophysiological Consequences of Sickle Cell Disease conference Nov. 6-8 in Washington, D.C. The world’s leading experts in the field of sickle cell disease (SCD) will present the…
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Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…
The Medical College of Georgia (MCG) and Augusta University Health (AU Health) have joined the first large, national clinical trial investigating bone marrow transplantation as part of the standard of care (SoC) for patients with sickle cell disease (SCD). The National Institutes of Health (NIH)-funded study will…
Hydroxyurea, a treatment recommended for children with sickle cell anemia (SCA), doesn’t increase the risk of malaria infection in sub-Saharan African countries where malaria is endemic, clearing up previous suspicions, a new study finds. Sold under the brand names Hydrea (Bristol-Myers Squibb) or Droxia (Bristol-Myers Squibb),…
Cydan, the company that founded Imara — which developed a sickle cell disease (SCD) treatment candidate, IMR-687 — has raised $34 million in new financing to advance innovative therapies for patients living with SCD and other rare diseases. Cydan, based in Cambridge, Massachusetts, identifies promising scientific findings and drug…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
The California Institute for Regenerative Medicine (CIRM) awarded Matthew Porteus, a Stanford University associate professor of pediatrics, a $5.2 million grant to conduct the groundwork leading to a clinical trial of a possible treatment for sickle cell disease (SCD). SCD develops when a gene is mutated that codes for a…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
The Medical University of South Carolina (MUSC) was recently awarded a Doris Duke Charitable Foundation grant to develop a new gene-modifying therapy for the treatment of sickle cell disease. The project, “Epigenetic Modulators for the Treatment of Sickle Cell Disease,” was awarded more than $750,000 as part of the…
The U.S. Food and Drug Administration (FDA) has awarded Dr. Kenneth Ataga, a $2 million grant over five years to support a prospective study in sickle cell anemia to determine biomarkers of specific cellular function changes in chronic kidney disease (CKD). Ataga, a researcher with the University of…
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