The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States
Spinal muscular atrophy (SMA) is a rare neuromuscular disease with a rapidly evolving treatment landscape. To better meet the needs of trial sponsors and the patient community in the United States (US) in this...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Ilse Peterson, Ros ángel Cruz, Fatou Sarr, Ann Marie Stanley and Jill Jarecki Tags: Position statement Source Type: research
More News: Brain | Clinical Trials | Internal Medicine | Neurology | Rare Diseases | Spinal Muscular Atrophy | USA Health