New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene therapy ...

Please provide your email address to receive an email when new articles are posted on . An investigational, fully human monoclonal antibody developed as a muscle-targeted therapy for spinal muscular ...

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...

Muscle loss (atrophy) due to inactivity is common after illness, injury, hospitalization or falls, and becomes increasingly frequent with aging. New research published in Advanced Science shows that ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. So most recently, there has been the ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

HOUSTON – A newly identified gene, atrogin-1, is involved in muscle loss associated with cancer, diabetes, fasting and kidney disease as well as in the atrophy occurring with disuse, inactivity, and ...

Scholar Rock Holding Corporation (NASDAQ:SRRK) President & CEO Jay Backstrom presented at the J.P. Morgan Healthcare Conference. The discussions mainly focused on apitegromab for spinal muscular ...

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...

Building on the recent U.S. FDA Fast Track Designation for AJ201 (Rosolutamide) and encouraging Phase 2 clinical results, AnnJi presented two scientific abstracts spanning translational transcriptomic ...