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 »
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 »
