Several experts are wondering how best to determine which spinal muscular atrophy (SMA) patients will benefit most from treatment with Spinraza…
Articles by Patricia Inacio, PhD
Genentech is working on the design of a Phase 3 clinical trial of olesoxime to address U.S. and European regulators’…
Children with spinal muscular atrophy type I (SMA-I) have a tough time performing pair-matching tests, which may be related to their poor…
During the 2015 Cure SMA Researcher Meeting, a number of novel studies focusing on Spinal muscular atrophy (SMA) were presented under the topic…
Isis Pharmaceuticals, Inc. has announced that it has initiated a Phase 3 clinical trial (CHERISH) that will test the safety…
Isis Pharmaceuticals, Inc. and AstraZeneca announced a new partnership to develop new, more efficient delivery methods of its…
PTC Therapeutics, Inc. announced that their collaborative program with Roche and the SMA Foundation (SMAF) for Spinal Muscular Atrophy…
A study entitled “Patient with spinal muscular atrophy with respiratory distress type 1 presenting initially with hypertonia” reports…
In the study entitled “Observational study of spinal muscular atrophy type I and implications for clinical…
In a recent study published in the Molecular Therapy journal, the authors report an improved method to deliver the…