First Patient Dosed in Phase 3 Trial of AVXS-101 in Presymptomatic Infants with SMA Types 1, 2 and 3
The first patient has been dosed in the Phase 3 clinical trial of AveXis’ gene therapy candidate AVXS-101 in presymptomatic…
Articles by José Lopes, PhD
#AAN2018 — RG7916 Works to Raise SMN Protein Levels in Babies with SMA Type 1, Trial Shows
A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called RG7916 was able to safely raise levels…
#AAN2018 — Type 1 SMA Babies Treated with AVXS-101 Achieving New Milestones, Trials Show
Type 1 spinal muscular atrophy (SMA) babies treated with AveXis’ gene therapy candidate AVXS-101 continue to achieve…
#AAN2018 – SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show
Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according…
Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports
Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar…
SMA Expert Reveals Insights into Patient Care, Hope for Future Cure in Interview
Spinal muscular atrophy (SMA) expert Basil T. Darras, MD, is optimistic about treatment advances and the possibility of a future…
FDA Grants Orphan Drug Status to SRK-015 for SMA
SRK-015 has received orphan drug status designation by the U.S. Food and Drug Administration…
AveXis Plans a May Start for Phase 3 European Trial of Its SMA Type 1 Gene Therapy
AveXis’ expects to start a Phase 3 clinical trial of its spinal muscular atrophy (SMA) gene therapy AVXS-101 in…
Pre-symptomatic SMA Infants Show Stable Improvements in Respiratory, Motor Function in Spinraza Clinical Trial
All 25 pre-symptomatic infants treated with Biogen’s Spinraza (nusinersen) in a Phase 2 clinical trial of spinal…
Lack of Key Transport Molecules May Lead to Nerve Fiber Loss in Neurodegenerative Diseases, Study Says
A disruption between two molecules that transport substances to the remote ends of special nerve fibers may result in neurodegenerative disorders…