Health Care Professionals - Neuromuscular and Neurodegenerative Disorders
Prosensa’s platform technology allows the development of RNA-modulating therapeutics that either interfere with splicing (exon skipping, exon inclusion, or splice mutation correction), remove mutant RNA, or block RNA expression, for different indications. Prosensa’s main focus is on neuromuscular and neurodegenerative disorders such as Duchenne Muscular Dystrophy, Myotonic Dystrophy and Huntington’s disease.
We currently focus on 3 Neuromuscular Disorders:
Duchenne Muscular Dystrophy
The severe and progressive deterioration of muscle fibers in Duchenne Muscular Dystrophy (DMD) is caused by the deficiency of dystrophin, a protein that is essential for the integrity of muscle fiber membranes.
Myotonic Dystrophy (DM1) is caused by expansion of an unstable trinucleotide repeat (CTG) in the 3’ untranslated region of the DMPK gene (Myotonic Dystrophy protein kinase).
Huntington’s disease (HD) is caused by expansion of an unstable trinucleotide repeat (CAG) in the 5’ untranslated region of the HTT gene.