Research
Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , […]
Read More ›Novartis Releases Update on LMI070 (Branaplam) Clinical Trial
Novartis has released the following update for the LMI070 (branaplam) clinical trial, on behalf of the program team. Dear SMA community, For years, our researchers […]
Read More ›2017 SMA Researcher Meeting Summary: Clinical Research Studies for 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 ›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 ›