Cytokinetics Presents Positive Clinical Data For SMA Drug Candidate
During the SMA Annual Conference Cytokinetics presented results from their Phase 1 study of CK-2127107, a novel small molecule activator of the fast…
Articles by Ana de Barros, PhD
BBrm02: BioBlast Pharma’s Novel Approach To Combat SMA
In a recent press release BioBlast Pharma Ltd., a clinical-stage orphan disease-focused biotechnology company, announced positive results of its preclinical…
Cure SMA Publishes New Booklet on Clinical Trials
An increasing amount of research on drugs to treat spinal muscular atrophy (SMA) is successfully progressing as…
Isis Pharmaceuticals Announces Results From Phase 2 Study of ISIS-SMNRx In Infants With SMA Type I
Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2…
Cure SMA Releases Updates On SMA Drug Discovery Programs
Cure SMA, a non-profit organization focused on supporting research projects to find a cure for spinal…
Over $100,000 Raised at New England Walk-n-Roll to Help SMA
Over 600 walkers and runners helped raised more than $100,000 during the 15th Annual New England Chapter Walk-n-Roll, on Saturday, May 16, at the DCR…
NIH Funds Cure SMA’s Researcher Meeting
The non-profit organization Cure SMA has been awarded a National Institutes of Health (NIH) grant…
Discussion Draft on Compassionate Drug Use for Disease Such as SMA Released
A 21st Century Cures discussion draft has been released by the U.S. House of Representatives Energy and Commerce Committee as a…
Gene Therapy Research Awarded Additional $445,000 Cure SMA Grant
Nonprofit organization Cure SMA has awarded a $445,000 grant to fund research on a gene therapy for the treatment of spinal…
PTC Releases Data On New SMA Drug
PTC Therapeutics recently announced the release of clinical data from their Phase I trial testing the effects of the company’s oral…