News

IMR-687 safely increases the levels of fetal hemoglobin and the number of fetal hemoglobin-producing cells in people with sickle cell disease (SCD), according to early data from an ongoing Phase 2a clinical trial. The findings support the continuation of IMR-687’s clinical development as a potential disease modifying…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

A smartphone app that measures hemoglobin levels using pictures of a person’s inner eyelid may help assess anemia in people with blood disorders such as sickle cell disease, a new study reports.

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…