Research
Cure SMA-funded Researcher Sara Custer Publishes Paper
Sara Custer, PhD, and her colleagues at Indiana University have published a new paper, “Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells” in the journal […]
Read More ›Roche Launches New Phase 2 Clinical Trials
Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, […]
Read More ›Catch Up on Research News from the 2016 Family Friendly Research Poster Session
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in […]
Read More ›Roche Provides New Update on Clinical Development of RG7916
Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA […]
Read More ›2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›2016 SMA Researcher Meeting Summary: SMA Therapy Development
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›Summer 2016 Issue of Compass Now Available
The Summer 2016 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced $704,000 in funding for […]
Read More ›2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
During the 2016 SMA Researcher Meeting, the anniversary of the identification of the SMN genes by Judith Melki MD, PhD (Inserm, Universite Paris) was recognized. […]
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