Global Genes recently published, “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process.” This toolkit was the product of a collaboration between Global Genes and leaders in advocacy and government, including Cure SMA. Work from Cure SMA was used as case studies in five different areas: encouraging research, funding […]
Global Genes Features Cure SMA in Patient Voice Project Read More »
Cure SMA has awarded a $30,000 research grant to Jocelyn Cote, PhD, at the University of Ottawa for his project, “The role of SMN as a translational regulator.” 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. We know this
Cure SMA Awards $30,000 Grant to Jocelyn Cote, PhD, University of Ottawa Read More »
Cure SMA has awarded a $140,000 research grant to Arthur Burghes, PhD, at The Ohio State University, for his project, “Defining the contribution of RNP assembly pathways to the SMA phenotype.” 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.
Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University Read More »
Cure SMA has awarded a $90,000 research grant to Antoine Cléry, PhD, at ETH Zurich for his project, “Novel approaches against spinal muscular atrophy by targeting splicing regulators.” SMA is caused by a mutation in the SMN1 gene, which regulates the production of survival motor neuron (SMN) protein. Individuals with SMA also have one or
Cure SMA Awards $90,000 Grant to Antoine Cl̩ry, PhD, ETH Zurich Read More »
Cure SMA has awarded a $140,000 research grant to Christine Beattie, PhD, at the Ohio State University, for her project, “Identification of SMN:HuD bound RNAs critical for motor neuron development.” 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, causing
Cure SMA Awards $140,000 Grant to Christine Beattie, PhD, Ohio State University Read More »
Cure SMA has released the schedule for the 2016 Annual SMA Conference, which will be held at Disneyland in Anaheim, CA, June 16-19. This schedule includes the timeline of workshops and events, including the Meet and Greet, Researcher Relay Race, Teen Social, Newly Diagnosed Program, Family Friendly Poster Session, and PJ Party & Movie Night.
Cure SMA Releases Schedule for the 2016 Annual SMA Conference Read More »
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 »
