Front Page News
SMA Newborn Screening Moves Forward in New Jersey
Earlier this month, the New Jersey State Senate Committee on Health, Human Services, and Senior Citizens unanimously approved S. 974. Introduced by Senator Troy Singleton, […]
Thank You For a Wonderful 2018 Annual SMA Conference!
Thank you to everyone who attended the 2018 Annual SMA Conference at the Hilton Anatole Hotel in Dallas, Texas! It was an impactful weekend of […]
Summer 2018 Issue of Compass Now Available
The summer 2018 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced ten grants totaling $1,325,000 […]
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 […]
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 John W. Day, […]
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 data–for […]
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 […]
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 […]
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 […]
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 enrollment of healthy […]