News

The European Commission has granted orphan drug designation to IMR-687, Imara’s potential new therapy for sickle cell disease (SCD). In the European Union, this designation is given to investigative therapies for rare diseases — those that affect fewer than five in 10,000 people — to…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to EDIT-301, a potential cell therapy for the treatment of sickle cell disease (SCD). This designation is given to therapies that aim to treat serious or life-threatening disorders that affect fewer than 200,000 people…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Use of medical marijuana to ease pain is associated with fewer visits to the hospital in people with sickle cell disease (SCD), a study suggests. Patients given medical marijuana felt this was an effective treatment for pain relief, and also reported a reduction in the need for opioids.

A Phase 2b clinical trial that is investigating the safety and effectiveness of IMR-687 in people with sickle cell disease (SCD) has dosed its first participant, according to the potential therapy’s developer, Imara. The study, Ardent (NCT04474314), intends to enroll almost 100 adults who have SCD. Recruitment…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The Scottish Medicines Consortium (SMC) has approved the use of Xromi (hydroxycarbamide), a liquid form of hydroxyurea, for treating children with sickle cell disease (SCD). Xromi is expected to be used in children older than 2 and younger than 9 who…

The cannabis company Parallel, which aims to improve quality of life with its products, has launched a 10-year collaboration with the University of Pittsburgh, known as Pitt, to investigate the potential of medical marijuana for treating people with sickle cell disease (SCD). Pitt’s research program will receive an…

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience.  Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…

The National Heart, Lung, and Blood Institute (NHLBI) awarded nearly $1 million to Athena Starlard-Davenport, a professor at the University of Tennessee Health Science Center (UTHSC), to support work into fetal hemoglobin as a potential way of treating sickle cell disease (SCD). “For the past 100 years,…