The Fall 2018 issue of Directions is now available online. Please visit our Support & Care Publications page to download this issue or past issues of Directions. Our biannual newsletter builds our community by sharing stories and experiences. Families from all over the world connect with one another through Cure SMA to organize, share photos, […]
Fall 2018 Issue of Directions Now Available Read More »
Cure SMA recently hosted a video contest to raise SMA awareness and provide the opportunity for teens and young adults (aged 12 to 21) to share their experiences. The purpose of this contest is to raise awareness about the impact that SMA has on teens and young adults, and the strategies used to help navigate
Congratulations to the Winners of Cure SMA’s Video Contest! Read More »
When Micah Biello was diagnosed with spinal muscular atrophy type 1, no one in his family knew about the disease. Now four years later, Micah’s parents, grandparents, and other family members lead a Cure SMA Walk-n-Roll team named Micah’s Milers. The family has rallied around Micah. Not only do they fundraise, but they also help
Community Spotlight: Rebecca Smith and Micah Biello Read More »
On January 7th, PTC Therapeutics, Inc. provided a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. Morgan Healthcare Conference. As part of the update, progress on regulatory filing for risdiplam was outlined. The risdiplam program is a collaboration between PTC, Genentech/Roche and SMA Foundation. The following
Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam Read More »
Are you or a family member between the ages of 12 and 25? Do you want to share your opinion on living with SMA and what is important to you regarding treatment options? In late November, Cure SMA launched the SMA Teen and Young Adult Clinical Meaningfulness Survey. Anyone with SMA between the ages of
Teen and Young Adult Survey Extended Through January 21st Read More »
It has been a little over two years since doctors told Chris and Kelly Jankowski that their first-born son, William, had spinal muscular atrophy (SMA) type I. In the immediate wake of diagnosis, Cure SMA was able to help provide the Jankowskis with the resources to help them make decisions about care: asking the best
Community Spotlight: The Jankowski Family Read More »
Cure SMA is pleased to announce the launch of an expanded Phase 3 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory
Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration Read More »
The 8th Annual “Hope on the Hill” Congressional Dinner was held on Tuesday, November 27, 2018 at The Willard Hotel in Washington, DC. More than 170 people from the SMA community – including government and industry partners – attended the dinner which raised $200,000 to fund advocacy efforts, research, and family support services. Since 2011,
Hope on the Hill Congressional Dinner Raises $200,000 Read More »
A recording of the December 13th webinar updating the community on SMA treatments and clinical trials is now available online. A PDF of the webinar presentation is also available for download. An update on the status of dosing for Spinraza across the United States included the following information: As of October 2018, ~2,700 patients have
December Webinar on SMA Treatments and Clinical Trials Now Available Online Read More »
Over our past fiscal year—from July 1, 2017, to June 30, 2018—Cure SMA has funded over $5 million in new research funding. This funding will be used strategically to help accelerate research, and ensure we are developing treatments for all types, ages, and stages of SMA. The areas funded include: Basic research, with a particular focus
2018 Research Year-in-Review Read More »
