Patent ID: 11872239
Assignee: SYNTHENA AG
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 8:
9. The composition of claim 1 for use as a medicament in the prevention, treatment or diagnosis of a disease, wherein the disease is a neuromuscular or musculoskeletal disease selected from Duchenne muscular dystrophy, familial dysautonomia, spinal muscular atrophy, ataxia telangiectasia, congenital disorder of glycosylation, fronto-temporal dementia, Parkinsonism linked to chromosome 17, Niemann-Pick disease type C, neurofibromatosis type 1, neurofibromatosis type 2, megalencephalic leukoencephalopathy with subcortical cysts type 1, Pelizaeus-Merzbacher disease, Pompe disease, myotonic dystrophy type 2 (DM2 or proximal myotonic myopathy), and myotonic dystrophy type 1 (DM1 or Steinert disease).