Today, AveXis, Inc. presented an interim analysis of data as of April 1, 2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, presented the data at the […]
The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them. During the event, families
Cure SMA Announces 2016 Family Friendly Poster Session Presenters Read More »
Recently, Cure SMA confirmed three new board members and several new committee members. These new members will provide additional leadership in the areas of advocacy, FDA/regulation, and research. We extend a warm welcome to new board members Annie Kennedy, Spencer Perlman, and John Porter, PhD. We also extend a warm welcome to new committee members
Cure SMA Welcomes New Board and Committee Members Read More »
Ionis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of nusinersen in infants with spinal muscular atrophy (SMA) at the American Academy of Neurology (AAN) meeting. The data reported show that there have been no new events, as defined by progression to permanent ventilation or death, in the study since December
Ionis Releases Data Update from Phase 2 Study in Infants with SMA Read More »
Cure SMA is featured by Astellas Pharmaceuticals as part of their new Changing Tomorrow Together website. Cure SMA Research Director Jill Jarecki contributed a spotlight piece about Cure SMA’s research model: how and why we fund different areas of research, and the crucial role our community plays in drug development for SMA. Because of the
Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals Read More »
One of the most significant aspects of the Annual SMA Conference is the bringing together of families and researchers in the same event. The 2016 Annual SMA Conference is poised to set a record for the largest conference ever, with well over 1100 families and researchers already registered. As the world’s largest gathering of SMA
Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development Read More »
Cure SMA-funded researcher Peter Schultz has been awarded a four-year NIH grant of just over $2 million to support his research into small molecules to treat SMA. Cure SMA has funded the work of Dr. Schultz and his team at the California Institute of Biomedical Research (Calibr) through three drug discovery grants in 2012 and
Cure SMA-Funded Researchers Receive NIH Grant Read More »
Cure SMA is pleased to announced that, thanks to a generous grant from The Dhont Family Foundation, we are offering conference scholarships to all adults with SMA. The scholarship covers registration for one adult, age 18+, and one aide. Each year, we look forward to reuniting as a community at the Annual SMA Conference. One
Adult Scholarships Offered for 2016 Annual SMA Conference Read More »
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 »
