June 19, 2018
PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings
On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy...
June 16, 2018
Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference
Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by...
June 12, 2018
The 2018 Annual SMA Conference, now in its 30th year, is set to kick off on June 14 and continue into a weekend full of programs and events that bring families, researchers and healthcare professionals together.
This year’s conference will be the largest in the Midwest to date....
June 12, 2018
Thank you to the following sponsors who are generously supporting the 2018 Annual SMA Conference. This one-of-a-kind event would not be possible without your support!
For more information about the below companies, please visit our conference sponsor...
June 11, 2018
Thank you to the following exhibitors who are generously supporting the 2018 Annual SMA Conference. This incredible and unique event would not be possible without your support!
For more information on any of the below companies, please visit our
June 7, 2018
As the summer season nears, many families will make plans to travel across the country to exciting destinations or to spend time with those they love. This is no different for those affected by SMA.
Although travel can present challenges, regardless of distance or method chosen, below...
June 1, 2018
Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...
May 31, 2018
The Ohio Newborn Screening Advisory Committee recommended that SMA be added to their state’s newborn screening panel. The recommendation now goes to the Ohio Department of Health for a rulemaking process. Once these rules are developed and approved, SMA will be officially added to the...
May 30, 2018
Roche today provided the following community statement on olesoxime.
Dear members of the SMA Community,
We would like to provide you with an update about olesoxime, an investigational molecule in SMA.
Since we bought olesoxime from Trophos in 2015, we have had many...