The European Medicines Agency has granted orphan drug designation to SLS-005 (trehalose) for the treatment of Sanfilippo syndrome. This designation, which follows a positive opinion from the agency’s Committee for Orphan Medicinal Products, was announced in a press release from Seelos…

Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose) — its investigational therapy for Sanfilippo syndrome — from the Committee for Orphan Medicinal Products (COMP), a part of the European Medicines Agency (EMA). Following this opinion from…

The Committee for Orphan Medicinal Products, a branch of the European Medicines Agency (EMA), favors designating SLS-005 (trehalose) an orphan drug as a potential treatment for Sanfilippo syndrome, Seelos Therapeutics, its developer, announced. The positive opinion will now be sent to the European Commission, which…

The European Medicines Agency (EMA) sent written guidance to Seelos Therapeutics, regarding a planned European study of SLS-005 (trehalose) as a treatment for Sanfilippo syndrome types A and B. Seelos will design its open-label, non-placebo controlled, Phase 2b/3 study based on…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…