Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy designed to boost motor function in people with spinal muscular atrophy (SMA). “With the strength of our Phase 3 data as the foundation of our submission, we look forward…

Two years of treatment with the investigational therapy apitegromab continued to improve motor function in Spinraza-treated children and young adults with later-onset spinal muscular atrophy (SMA) who cannot walk, according to new extension data of the Phase 2 TOPAZ trial. The therapy’s developer, Scholar Rock, is also…

Treatment with the experimental muscle-directed therapy apitegromab improved motor function for many of the children and young adults with types 2 and 3 spinal muscular atrophy (SMA) after one year in the Phase 2 TOPAZ trial. Trial results were presented at the 2022 annual meeting of the American…

SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal muscular atrophy (SMA) throughout 2021. As a reminder of what mattered most to you during this year, here are the top 10 most-read articles of 2021 with a brief description of…

Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and young adults with types 2 and 3 SMA who are unable to walk. Apitegromab will be given as an add-on therapy to…

Scholar Rock is on track to initiate a pivotal Phase 3 trial of apitegromab in non-ambulatory patients with type 2 and type 3 spinal muscular atrophy (SMA) by the end of 2021, according to a company press release. The planned randomized, double-blind, and placebo-controlled trial will focus on people…

Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for spinal muscular atrophy (SMA), in type 2 and 3 patients who are unable to walk. “We anticipate initiating a Phase 3 trial to evaluate apitegromab in patients with non-ambulatory Type…

A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient trials, a study reported. The study, “Preclinical Safety Assessment and Toxicokinetics of Apitegromab, an Antibody Targeting…

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 towards our aim of establishing apitegromab as…

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 a Phase 1 clinical trial. These findings, published in the journal Advanced…