Progressive muscular atrophy (PMA) is clinically characterized ... in ALS included scores on the revised ALS Functional Rating Scale (ALSFRS-R), and pulmonary forced vital capacity.

Study using a new, child-friendly scale found patients up to age 2 had smaller mouth openings than healthy peers, risking ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...

who can experience atrophy in as little as 24 hours, says Fabrisia Ambrosio, a stem cell biologist in physical medicine and rehabilitation at Harvard University who was not involved in the study. The ...

Assessing the therapy’s efficacy and safety using the Revised Upper Limb Module (RULM) scale were the secondary ... "Novartis’ Phase III spinal muscular atrophy therapy trial meets primary ...

There is no cure for spinal muscular atrophy, but treatments can stop its ... "While I appreciate the large-scale review of this policy overall, I think in Jeremy's individual case — which ...