Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases

Leave a Comment / Front Page News, Research /

Current knowledge regarding clinical meaningfulness and quality of life amongst teens and young adults with spinal muscular atrophy (SMA) is limited. Much of the available qualitative data on this population has been obtained from the perspective of the parents/caregivers rather than the individuals themselves. Knowing this, Cure SMA is pleased to announce the publication of a manuscript, […]

Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases Read More »

Community Spotlight: I am…Viola Dwyer

Leave a Comment / Community Spotlight, Front Page News /

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we have been posting stories about SMA community members, showcasing their greatest passions, hobbies, and tidbits of their life. Viola Dwyer is from a suburb of Philadelphia and recently pivoted from her corporate job in the financial sector to creating a

Community Spotlight: I am…Viola Dwyer Read More »

Jessica’s Story: Remembering Piper Grace

Leave a Comment / Community Spotlight, Front Page News /

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative—we will be posting stories about SMA community members, showcasing their greatest passions, hobbies, and tidbits of their life. This guest post is written by Jessica Hoefler whose daughter, Piper, passed away in May 2020, shortly after being diagnosed with SMA

Jessica’s Story: Remembering Piper Grace Read More »

Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM

Leave a Comment / Clinical Trials, Front Page News, Research /

Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy (SMA) were published in the February 24, 2021 online issue of the New England Journal of Medicine (NEJM). The data show

Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM Read More »

Cure SMA Launches Virtual Therapy Program for Adults with SMA

Leave a Comment / Events & Fundraising, Front Page News, Support & Care /

Adults with spinal muscular atrophy (SMA) are invited to take advantage of Cure SMA’s latest support resource, the Adults with SMA Virtual Therapy Program, which has been generously sponsored by Genentech and the Dhont Family Foundation. The Adults with SMA Virtual Therapy Program provides adults with SMA three complementary, 30-minute live video sessions with a

Cure SMA Launches Virtual Therapy Program for Adults with SMA Read More »

Community Spotlight: I Am…Kelsey Roberts

Leave a Comment / Community Spotlight, Front Page News /

February 28 is Rare Disease Day. Throughout the month—in recognition of our Redefining Rare initiative — we will be posting stories about SMA community members, showcasing their greatest passions, hobbies and tidbits of their life. Kelsey Roberts is a determined, kind, and passionate 18-year-old from Massachusetts. As a teenager, she was diagnosed with spinal muscular

Community Spotlight: I Am…Kelsey Roberts Read More »

Cure SMA Career Panel Webinar Series to Launch in February

Leave a Comment / Events & Fundraising, Front Page News, Support & Care /

Cure SMA is excited to invite all members of the SMA community to join our new, three-part Career Panel Webinar Series, generously sponsored by Biogen. During these 1-hour webinars, adults with SMA will share honest and insightful perspectives into their career journeys—from accomplishments to challenges and everything in between. Attendees will be able to submit

Cure SMA Career Panel Webinar Series to Launch in February Read More »

Cure SMA Announces 2021 Summit of Strength Program Events

Leave a Comment / Events & Fundraising, Front Page News, Support & Care /

Cure SMA is excited to share that we will be continuing the Summit of Strength Program in 2021, offering a hybrid of the original in-person events and the webinars launched in 2020. This year marks the program’s fourth year, having brought together nearly 3,000 attendees from across the U.S. in-person, and close to 1,300 live

Cure SMA Announces 2021 Summit of Strength Program Events Read More »

Scroll to Top