Mothers of SMA Patients Feel Better After Engaging in Social and Leisure Activities, Study Says
More frequent participation in social and leisure activities is associated with less caregiver burden and emotional distress for mothers of…
Articles by José Lopes, PhD
Speech-generating Devices Provide Benefits for Children with SMA Type 1, Parent Survey Shows
Use of speech-generating devices (SGDs) improves the quality of life of children with spinal muscle atrophy (SMA) type 1 through…
New Gene Therapy Approach Able to Repair Mutations Causing SMA, Other Inherited Diseases, Mouse Study Suggests
A new gene therapy approach using RNA molecules called transfer RNAs (tRNAs) was able to repair a subset of…
FDA OKs 6MWT as Primary Goal of Potential Reldesemtiv Program for SMA Patients
The U.S. Food and Drug Administration approved the six-minute walk test (6MWT) as an acceptable primary endpoint, or goal,…
Zolgensma Could Be More Cost-effective than Spinraza with One-time $2 Million Price, Report Says
A preliminary Draft Evidence Report from the Institute for Clinical and Economic Review (ICER) says the investigational spinal…
Top 10 SMA Stories of 2018
SMA News Today brought you daily coverage of important findings, key treatment developments and clinical trials related to spinal muscular atrophy…
Post-Risdiplam Blood Test Could Assess SMA Patients’ Body-wide Protein Levels
An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect…
Experienced, Multidisciplinary Teams Can Ensure Success of Spinraza Programs, Study Reports
Treatment of spinal muscular atrophy (SMA) with Spinraza (nusinersen) through the spinal canal is feasible and leads to…
Zolgensma (AVXS-101) Under Priority FDA Review as Possible Gene Therapy for SMA Type 1
The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that…
Possible $4M Price for AVXS-101, ‘Foundational’ SMA Gene Therapy, Could Be Cost-Effective, Novartis Says
Novartis may put a $4 million to $5 million price tag on its “one-time, potentially curative” gene therapy AVXS-101…