Front Page News
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 […]
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. […]
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. […]
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. […]
2016 SMA Researcher Meeting Summary: Clinical Research Studies
We will be posting a series of summaries from our 2016 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Cure SMA Plans a Series of Educational Modules for the SMA Community
With the recent news that Biogen and Ionis will submit nusinersen for FDA approval—along with other recent developments, including the breakthrough designation for AveXis’s gene […]
Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
Dr. Chris Lorson and his colleagues have published a paper, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy,” in […]
AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
AveXis recently released new data from the Phase 1 clinical trial of AVXS-101, a gene therapy program to treat SMA. The interim data through July […]
2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
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. […]
Novartis Releases Community Update on the Study of LMI070
Novartis recently provided a community update on clinical trials for LMI070. This information is effective as of July 25 2016: “In May, we shared with […]