AveXis has released new data from their current Phase 1 clinical trial testing AVXS-101, a systemic delivery of gene therapy for SMA. The data from this initial trial will inform future clinical studies of gene therapy approaches. “The past six months has been a period of significant progress for AveXis, as we completed a successful […]
AveXis Releases New Data on Phase 1 Clinical Trial Read More »
Abstract submission is now closed for the 2016 SMA Researcher Meeting. We are excited to announce that we received 118 submissions, a record number. Our submissions come from researchers in 14 countries, six companies, and over 50 institutions. We expect over 300 researchers and clinicians from almost 100 organizations to attend the SMA Researcher Meeting.
Record Number of Abstracts Submitted for the 2016 SMA Researcher Meeting Read More »
The Senate Health, Education, Labor and Pensions (HELP) Committee yesterday passed an additional 7 bills out of committee. Along with the 7 bills previously passed out of committee, that makes 14 bills in total that have passed out of committee as part of the Senate’s “step-by-step” approach to Innovation and 21st Century Cures. Among the
Senate HELP Committee Passes Another 7 Innovation Bills Read More »
The Winter 2015 issue of Directions is now available! 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 each other through Cure SMA to chat, organize, and share
Winter 2015 Issue of Directions Now Online Read More »
Cure SMA-funded researcher Chien-Ping Ko has published a paper in the Journal of Neuroscience, called “Defects in Motoneuron–Astrocyte Interactions in Spinal Muscular Atrophy.” The research for this paper was supported by a donation from the Dhont Family Foundation to Cure SMA. Individuals with SMA don’t correctly produce survival motor neuron (SMN) protein at high enough
Cure SMA-Funded Researchers Publish Paper on Motor Neuron and Astrocyte Defects Read More »
I am a medical education researcher fielding a survey to individuals or caregivers of individuals with Type II or III spinal muscular atrophy (SMA) to understand their specific challenges as well as informational and educational needs. The purpose of the research is to develop a report to inform supporters of patient education of the real-world
Registration for the Annual SMA Conference is well on its way to a record-setting number. Over 700 families and researchers have registered for the conference thus far—the highest number to this point in over five years! We are excited to have so many new and familiar faces joining us in June. If you have not
2016 Annual SMA Conference on Track to Break Attendance Records Read More »
Cure SMA has awarded a $140,000 research grant to Rashmi Kothary, MD, at the Ottawa Hospital Research Institute for his project, ” Muscle satellite cell biology and muscle regeneration in Smn-depleted mice.” Because of a genetic mutation, individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels. We know that
Cure SMA Awards $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute Read More »
Cure SMA has awarded a $140,000 research grant to Megerditch Kiledjian, PhD, at Rutgers, The State University of New Jersey, for his project, “Novel Strategies to Increase SMN2 RNA.” Because of a genetic mutation in the SMN1 gene, individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels. All individuals
Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers Read More »
The Senate Health, Education, Labor and Pensions (HELP) Committee has passed 7 bills out of committee as part of its “step-by-step” approach to Innovation and 21st Century Cures. Among them is The Advancing Research for Neurological Diseases Act of 2015 (S.849), sponsored by Sens. Isakson (R-Ga.) and Murphy (D-Conn.), which would expand research on neurological
Senate HELP Committee Passes Innovation Bills Read More »
