Muscle-directed Therapy Apitegromab Put on FDA’s Fast Track
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was granted a fast track designation by the U.S. Food and Drug Administration. “We are delighted to receive Fast Track designation and look forward to working closely with the FDA...
Improving Muscle Length to Prepare For Future SMA Muscle Targeted Therapy
In preparation for a muscle-targeted treatment, which is currently under development by Scholar Rock for patients with SMA, we have continued to work to maintain the strength improvements Michael has made, but we have also shifted to increasing his range of motion in...
SMN2 Copy Number and Rare Variant Influence SMA Severity
The number of copies of the “backup” SMN2 gene and a rare genetic variant in this gene influence disease severity in people with spinal muscular atrophy (SMA), according to data from more than 2,000 people involved in a free SMA genetic testing program. Launched in...
SMA May Carry Higher Risk of Fluid Buildup in Brain, Study Finds
Spinal muscular atrophy (SMA) patients may at a fourfold greater risk of hydrocephalus, or abnormal accumulation of cerebrospinal fluid (CSF) in the brain, than their healthy peers, a large U.S. health record-based study suggests. Data also showed that patients...
Apitegromab Safely Counters Muscle Growth Suppressor, Phase 1 Trial Finds
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance of the inactive form of myostatin, a muscle growth suppressor, in healthy volunteers, according to data from...HCP — SMA News Today
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