FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an orphan drug by the European Medicines Agency (EMA). The decision was based on a positive recommendation by EMA’s Committee for Medicinal Products for Human Use. Orphan designation is given to investigative…
A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant to build clinical and research infrastructure that will improve diagnosis and treatment for children living with sickle cell disease (SCD) in Malawi. Malawi is a country located in sub-Saharan Africa. Each year…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), CSL Behring, the medicine’s maker, announced in a press…
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval covers the use of Adakveo in combination with hydroxyurea (also known as hydroxycarbamide) — another approved therapy…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not yet enrolling, aims to recruit 88 adults at its single Altasciences Clinical Kansas site. The…
Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell disease — is now available in 100 mg scored tablets, in addition to the 1,000 mg triple-scored tablet form. Scored tablets make measuring medicine more flexible, giving patients greater control in finding…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for sickle cell disease (SCD), after the treatment failed to show adequate activity in a Phase 2 clinical trial, the company announced. The STRONG-SCD (NCT03285178), which completed patient enrollment…
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