Myostatin-Targeting Biologic Fast Tracked for Spinal Muscular Atrophy
The Company is currently evaluating the efficacy and safety of taldefgrobep alfa in patients with SMA in the phase 3 RESILIENT study.
Diagnosis & Disease Information
Risdiplam Gets Prority Review for Presymptomatic Spinal Muscular Atrophy
Risdiplam is a survival motor neuron 2 splicing modifier.
Amyotrophic Lateral Sclerosis Prevention: Neurodegenerative Diseases Offer Insight
Researchers sought to develop a “roadmap” to early intervention and disease prevention for ALS by observing advances in neurodegenerative diseases with a presymptomatic period.
Zolgensma Labeling Updated With Additional Safety Information
Zolgensma is an adeno-associated virus vector-based gene therapy.
FIREFISH Trial Shows Risdiplam Benefits in Infants With Type 1 Spinal Muscular Atrophy
Researchers evaluated the safety and efficacy of risdiplam in infants with type 1 spinal muscular atrophy compared with historical cohorts.
Apitegromab Fast Tracked for Spinal Muscular Atrophy
Apitegromab is a selective inhibitor of the inactive form of myostatin to block its activation in the muscle.
- Loading...
Loading...
Next post in News
Loading... 
Loading...