News

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

Howard University researchers have discovered a link between increases in Veillonella gut bacteria and higher levels of pain in sickle cell disease patients. The team presented their findings at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, Nov. 6-8. Sickle cell disease is a blood…

Global Blood Therapeutics (GBT) reported a case study in which a sickle cell disease (SCD) patient was granted compassionate access to the company’s ongoing Phase 3 HOPE study of voxelotor (NCT03036813). The patient had severe, transfusion-refractory anemia and experienced rapid improvements with the therapy.

Memantine, a standard treatment for Alzheimer’s disease, has shown promise in treating sickle cell disease (SCD) in six patients, a 12-month pilot clinical trial found. The findings were presented at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, D.C. The study’s lead researcher,…

Sancilio Pharmaceuticals says its investigational therapy Altemia (SC411) achieved primary and secondary endpoints in a clinical trial with sickle cell patients 5 to 17 years old. Altemia is a combination of specific lipids designed to restore balance to the membrane of red blood cells that are damaged in sickle…

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…

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…