AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported topline results from the Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1. The company also reported financial results for the fourth quarter and full year ended December 31, 2016, recent
AveXis Reports Results from Phase 1 Trial of AVXS-101 Read More »
Your elected officials who represent you in Washington, D.C. have caseworkers in their regional and district offices in your communities. These caseworkers can help you work with and better understand government programs, like Medicaid, Medicare, TRICARE, and Social Security. Caseworkers, also sometimes called constituent services representatives, are in the local community offices of federal elected
Cure SMA has awarded a $140,000 research grant to Arthur Burghes, PhD, at The Ohio State University, for his project, “Identification of SMA modifiers and deletion/duplication junctions in the SMA region.” All individuals with SMA lack the SMN1 gene, but have one or more copies of SMN2, the SMA “backup gene.” In general, those with
Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University Read More »
Cure SMA is excited to announce the launch of our newest care series booklet, Choice and Connection to Care: A Health Insurance Roadmap for People Living with Spinal Muscular Atrophy (SMA) and Their Caregivers. You can download a copy of this booklet now from our website, or send an email to [email protected] to request a
Cure SMA Releases Care Series Booklet on Insurance Read More »
Cure SMA and the SMA Newborn Screening Coalition are excited to announce we have completed the submission of SMA to the Recommended Uniform Screening Panel (RUSP). Administered by the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC), part of the Department of Health and Human Services, the RUSP is a list of
SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP) Read More »
Just added to this year’s conference schedule: Saturday night at Magic Kingdom Park! We are excited to announce that each conference attendee will get to participate in this classic Disney World evening, sponsored by Biogen. All registered participants will receive complimentary Saturday Disney Entrance to Magic Kingdom Park, and the opportunity to watch the Happily
Announcing an Evening of Wonder at Magic Kingdom Park Read More »
Cure SMA is pleased to announce a new webinar on interactions between the FDA and the SMA community. The webinar will be held on March 8, 2017, at 1:00pm EST (10:00am PST/ 11:00am MST/ 12:00pm CST). The goal is to provide both a broad overview of how our community can continue to productively engage with
Cure SMA to Host Webinar with the FDA and Patient Advocates Read More »
Registration is now open for the SMA Patient-Focused Drug Development (PFDD) meeting with the FDA. The PFDD meeting will be held at the College Park Marriott, near Washinton DC, on Tuesday, April 18. There are two primary ways to participate in this meeting: Attend in person. Registration for in-person attendance is free, but seating is
Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA Read More »
On Tuesday, February 14, at 1:00pm EST (10:00am PST/11:00am MST/12:00pm CST), Cure SMA hosted a webinar updating the community on the status of Spinraza access. Among the topics covered were adminstration sites, insurance coverage, and community resources and support programs. We were happy to host an open, candid, and informal discussion of where our community
Spinraza Access Update Webinar Read More »
For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily life with SMA and the ongoing
