Research
Fall 2017 Issue of Compass Now Available
The fall 2017 issue of Compass is now available online. This issue covers Phase II of the SMA Industry Collaboration, as well as highlights and […]
Cure SMA Announces Over $5 Million in New Research and Care Funding
Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help […]
Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)
Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the […]
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 , […]
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 […]
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 […]
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 […]
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 […]
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 […]
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 […]