GENE ONLINE|News &
Opinion
Blog

Spotlight: Roche’s SMA Drug, Risdiplam Makes Strong Case for Regulatory Approval

by GeneOnline
Share To
Spinal muscular atrophy (SMA), a motor neuron disease, is the leading inherited cause of death in newborns, affecting approximately one in 10,000 individuals. In SMA patients, recessive loss-of-function mutations in the survival motor neuron 1 gene (SMN1) results in a reduced amount of functional SMN protein, which then leads to loss of α motor neurons and severe muscle weakness, particularly in proximal, truncal, and bulbar muscle groups. In patients with the most severe infantile-onset form (type I), infants do not survive beyond the first two years of life due to progressive hypotonia,

GO Prime with only $1.49 now

LATEST
Novartis’ CDK4/6 Inhibitor Meets Phase 3 Endpoints In Breast Cancer
2023-03-28
BD and A*STAR collaborate to Advance Ultra-High Dimensional Analysis in Flow Cytometry for Immunology Research
2023-03-28
Vertex and CRISPR Collaborate Again with $330 Million Diabetes Gene Editing Deal
2023-03-28
Amway Applauded by Frost & Sullivan for Capitalizing on Innovative Competitive Strategies to Drive Differentiation in Its Home Water Treatment Solution in APAC
2023-03-28
BJ Bioscience Announces Clinical Collaboration with MSD to Evaluate BJ-001 in Combination with KEYTRUDA® (pembrolizumab)
2023-03-27
35% Of Better Therapeutics’ Staff to be Cut to Extend the Company’s Runway
2023-03-27
Pharming’s Joenja Racks Up FDA Approval For Rare Primary Immunodeficiency
2023-03-27
Scroll to Top