We are excited to announce that registration is now open for the 2020 Annual SMA Conference, which will be held at Disney’s Yacht & Beach Club Resort in Orlando, Florida from Thursday, June 11 – Sunday, June 14, 2020. We hope you can join us for another magical weekend together in Disney World! With more […]
Registration Now Open for the 2020 Annual SMA Conference! Read More »
David Alvarez is a 19-year-old student from Seymour, Conn., majoring in Business Management/Administration and Marketing at the University of Bridgeport. He is also the founder of MADE Clothing, a business venture he began in high school to empower and remember those who have “made it” (or are currently “making it”) through their personal struggles. This
Community Spotlight: David Alvarez Read More »
The 2019 Summit of Strength Program was a huge success, with more than 135 speakers sharing their expertise on numerous highly rated and unique topics. We would like to thank these wonderful speakers and all attendees for making this year’s program so engaging and insightful. Summits of Strength are single-day, educational events providing information for
Update on Cure SMA Summit of Strength Program – Gearing Up for 2020! Read More »
There are now two treatments approved by the U.S. Food and Drug Administration (FDA) that target the underlying genetics of spinal muscular atrophy (SMA) and several others in clinical trials. The advent of these new treatments for SMA has led to community discussion around the possibility of combining therapies in the hopes of better outcomes.
Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations” Read More »
Just one more week until the 9th Annual “Hope on the Hill” Congressional Dinner in Washington D.C.! Next Tuesday, December 3rd, SMA families and advocates, government officials, and industry leaders will gather to discuss advancements in research, treatment, and patient care, as well as harness the latest momentum to set the course for the next
9th Annual “Hope on the Hill” Congressional Dinner Read More »
Cure SMA is thrilled to announce that we will, once again, be offering scholarship opportunities to adults with SMA (18 years of age and older) to attend the 2020 Annual SMA Conference. The conference will take place June 11-June 14, 2020, at Disney’s Yacht & Beach Club Resort in Orlando, FL. Thanks to a generous
Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA). If approved, risdiplam, an orally administered liquid, would be the first at-home
Earlier this month, an article was published in the Journal of Neuromuscular Diseases titled, “Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings from the Cure SMA Cohort.” This paper examined the concordance and discordance of SMA sub-type among siblings in order to learn about the family experience of SMA, support the identification of genetic
SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases Read More »
Many consider the deepest expression of love is being a caregiver. Providing for someone’s needs is incredibly special and rewarding to the one who is giving, as well as the one receiving. Despite this, the extra demands placed on caregivers can often lead to chronic stress and stress-related disorders. Chronic stress, if left untreated, can
Caring for the Caregiver by Brynne Willis Read More »
Scholar Rock today announced preliminary pharmacokinetic (PK) and pharmacodynamic (PD) results from the TOPAZ Phase 2 proof-of-concept trial of SRK-015 for the treatment of patients with spinal muscular atrophy (SMA). The planned preliminary PK/PD analysis, which includes data from 29 patients with SMA across all three cohorts, showed dose-proportional drug exposure and demonstrated target engagement, as
