News
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...
Topics: Clinical Trials, Research, Front Page News
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...
Topics: Clinical Trials, Research, Front Page News
June 14, 2018
Biogen to Present New Data at the 2018 Annual SMA Conference
Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of...
Topics: Conference, Research, Front Page News
June 12, 2018
The 2018 Annual SMA Conference is Here!
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....
Topics: Conference, Front Page News
June 12, 2018
Thank You to Our 2018 Annual SMA Conference Sponsors!
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...
Topics: Conference, Front Page News
June 11, 2018
Thank You to Our 2018 Annual SMA Conference Exhibitors!
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
Topics: Conference, Front Page News
June 7, 2018
Traveling with SMA
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...
Topics: Community & Awareness, About SMA, Front Page News
June 1, 2018
Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing...
Topics: Clinical Trials, Research, Front Page News
May 31, 2018
Ohio Moves One Step Closer to SMA Newborn Screening
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...
Topics: Our Impact, Advocacy, Front Page News
May 30, 2018
Roche Releases Community Statement on Olesoxime Program
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...
Topics: Clinical Trials, Research, Front Page News
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