News

Anyah Randolph, a teenager with sickle cell disease (SCD), was recently able to join her school class on a trip to Europe – and even climb the Eiffel Tower in Paris after the elevator broke – thanks to the help of hydroxyurea and Loyola Medicine pediatricians. Anyah had…

Following a review of studies published in the last decade, experts have updated their recommendations for managing a sickle cell disease-related eye complication known as sickle cell retinopathy (SCR). Their paper, “Sickle cell retinopathy: improving care with a multidisciplinary approach,” appeared in the Journal of Multidisciplinary Healthcare. SCR, a…

An independent review board has found Global Blood Therapeutics’ sickle cell disease treatment voxelotor safe, and recommended that its Phase 3 clinical trial continue. The hallmark of sickle cell disease is damage to a red blood cell protein called hemoglobin that carries oxygen to organs and tissue. The damage causes the cells…

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…