Research
2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers
The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]
Read More ›Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field
During the 2017 SMA Researcher Meeting, we honored one of our 2017 conference champions, Dr. Victor Dubowitz. Dr. Dubowitz’s early contributions to the SMA field […]
Read More ›Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers
Two Cure SMA-funded projects have recently been published. Diane Murrell and her team published the paper, “The Experience of Families With Children With Spinal Muscular […]
Read More ›2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression
The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]
Read More ›2017 SMA Researcher Meeting Summary: SMA Therapy Development
The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]
Read More ›2017 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA
The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 […]
Read More ›Summer 2017 Issue of Compass Now Available
The summer 2017 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1.03 […]
Read More ›Summer 2017 Update to SMA Drug Pipeline is Released
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 19 active programs, including one approved therapy. 14 pharmaceutical partners. 5 […]
Read More ›Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal
The journal Neuromuscular Disorders has published a paper reporting on the findings of an international workgroup convened to update the Standard of Care (SOC) for […]
Read More ›AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101
AveXis today announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following […]
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