Neuromyelitis optica (NMO) can be a devastating rare disease, but a lack of awareness among doctors means patients are often misdiagnosed for years, while research into cures is sparse.
Novartis’ AveXis unit has said European regulators have approved its Zolgensma (onasemnogene abeparvovec) gene therapy for the rare childhood wasting disease spinal muscular atrophy (SMA),
Japan’s Takeda has posted a surprise profit after its $59 billion merger with Shire just over a year ago, as the company shakes off the huge debts it incurred to get the deal done.
With trial delays, social distancing, and falls in charitable funding, the coronavirus pandemic presents some serious challenges for nonprofit orphan disease biotechs like Cure Rare Disease
