Scotland begins testing newborn babies for rare genetic condition

Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...
New Atlas

Protein linked to muscle development could be new atrophy treatment target

Researchers have identified a protein that’s critical to the development of skeletal muscle mass and strength and the tissue’s ability to metabolize glucose. They say their findings may lead to ...
Healio

Electrical stimulation device may mitigate facial muscle atrophy from botulinum toxin

Please provide your email address to receive an email when new articles are posted on . Patients in the active treatment group saw a 37.4% reduction in wrinkle severity from baseline. They also saw a ...
Medscape

Start Spinal Muscular Atrophy Treatment at Birth?

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 ...

Making space for everyday life with spinal muscular atrophy

But for Brooklyn, who lives with spinal muscular atrophy, or SMA, a rare genetic condition that affects muscle strength and ...
Business Wire

Abcuro Initiates Part B of Registrational Phase 2/3 MUSCLE Clinical Trial of Ulviprubart (ABC008) for the Treatment of Inclusion Body Myositis

NEWTON, Mass.--(BUSINESS WIRE)--Abcuro, Inc., a clinical-stage biotechnology company developing therapies for the treatment of autoimmune diseases and cancer through precise modulation of cytotoxic T ...
Science Daily

New target found for treatment of spinal muscular atrophy

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...
Seeking Alpha

FDA Issues Complete Response Letter (CRL) for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at Catalent ...

Scholar Rock (SRRK) intends to resubmit the apitegromab Biologics License Application (BLA) upon resolution of Catalent Indiana LLC-related observations CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar ...
Seeking Alpha

FDA Grants Priority Review for Biologics License Application (BLA) and EMA Accepts Marketing Authorisation Application (MAA) for Apitegromab as a Treatment for Spinal Muscular ...

Apitegromab Marketing Authorisation Application to the European Medicines Agency validated and under review CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock (SRRK), a late-stage biopharmaceutical ...

Scotland becomes first part of UK to screen babies for rare muscle disease

Scotland becomes first part of UK to screen babies for rare muscle disease - All parents will now be offered spinal muscular ...