Advancium Health Network, a public charity dedicated to advancing therapies for rare diseases, has awarded two major grants ...

New CMTA-STAR project aims to uncover elusive mutations in CMT through long-read sequencing, helping more patients reach a genetic diagnos ...

Bamboo has pre-clinical treatments for Duchenne muscular dystrophy, Friedreich’s Ataxia and Canavan disease, and a phase 1 asset for Giant Axonal Neuropathy. Unlike Biogen and its investment of ...

Neurological assessment comprised nerve conduction studies (NCS) and magnetic resonance imaging (MRI). NCS indicated axonal neuropathy, and MRI revealed T2/FLAIR hyperintensities in the dentate nuclei ...

Multiple myeloma is part of the spectrum of diseases ranging from MGUS to plasma cell leukemia, and similar to MGUS, is characterized by proliferation of a plasma cell clone and subsequent ...