News

Taking vitamin D supplements may lower the number of visits to the emergency department due to vaso-occlusive pain episodes in African American children with sickle cell disease (SCD), a study found. The findings also showed that 45% of these children with SCD have vitamin D deficiency. The study, “…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.

Bluebird Bio extended a partnership with two divisions of Hitachi Chemical for the long-term development and manufacturing of LentiGlobin, its investigational gene therapy for sickle cell disease (SCD), and other potential and approved treatments. The two subsidiaries — Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma — will be in charge…

Hemanext, a medical technology company, is working with the Sickle Cell Disease Association of America (SCDAA) to help people with sickle cell disease through a mix of educational programs, grassroots events, and public-awareness campaigns. “During these very…

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…

Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat (AG-348) as a potential treatment of sickle cell disease. Mitapivat is in an ongoing proof-of-concept Phase 2 trial in up to 25 patients with stable SCD, but further enrollment has…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The announcement was made by CRISPR Therapeutics and Vertex…

An advisory committee of the Sickle Cell Disease Association of America is recommending that people with sickle cell disease (SCD) be considered a “vulnerable” population during the reopening of the U.S. economy, and remain at home as much as possible. Patients should also continue taking their medications, and adopt…