Ionis Pharmaceuticals recently presented an update of its ongoing open-label Phase 2 clinical study of nusinersen, often called Ionis-SMN, in infants with spinal muscular atrophy (SMA) at the 2016 American Academy of Neurology meeting. According to the results, the investigational compound increased event-free survival, muscle function, and neuromuscular physiology, while no safety and tolerability…
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FightSMA and The Gwendolyn Strong Foundation (GSF), two organizations dedicated to ending spinal muscular atrophy (SMA), have announced three winners of their 2016 Emerging Investigator Awards (EIA). The winners of the EIA competition were announced at the Annual FightSMA Research Conference April 7-9 in…
FightSMA, an all-volunteer, parent-led nonprofit dedicated to finding treatments for spinal muscular atrophy (SMA), celebrated its 25th anniversary with a research conference that attracted leading SMA researchers from all over the world and received financial support from a record number of corporate sponsors. The 2016 FightSMA Annual Research…
Tariq Rahman, Ph.D., a research scientist at the Alfred I. DuPont Hospital for Children in Wilmington, Delaware, has been awarded a $50,000 Cure SMA clinical care research grant for his project, “Outcome Measures Using WREX – An Upper Extremity Exoskeleton for Children with SMA.” The project will analyze one particular…
Global Genes, a leading rare disease patient advocacy organization, recently published a new toolkit titled “From Molecules to Medicines: How Patients Can Share Their Voices Through the Drug Development Process” to address the challenges of patient participation in drug development. According to a press release, the toolkit…
As a part of Cure SMA’s basic research funding, Arthur Burghes, Ph.D., from The Ohio State University, has been awarded $140,000 for his project, “Defining the contribution of RNP assembly pathways to the SMA phenotype.” Burghes’ research will focus on the cellular function of survival motor neuron (SMN) proteins in…
Cure SMA has awarded a $90,000 grant to Antoine Cléry, Ph.D., a researcher at ETH Zurich, for his work on spinal muscular atrophy (SMA), titled “Novel approaches against spinal muscular atrophy by targeting splicing regulators.” The award is part of an $890,000 new basic research fund that…
Cure SMA has just awarded Ohio State University’s Christine Beattie, Ph.D., a $140,000 basic research funding grant for her project on spinal muscular atrophy (SMA): “Identification of SMN:HuD bound RNAs critical for motor neuron development.” Beattie is a professor in OSU’s Department of Neuroscience. She began studying motor…
AveXis recently reported favorable results from an ongoing Phase 1 clinical trial examining its candidate, AVXS-101, for the systematic delivery of gene therapy to young patients with spinal muscular atrophy (SMA). These preliminary results will help inform upcoming trials on gene therapy approaches. The open-label, dose-escalation study evaluating the safety and efficacy of…
RaNA Therapeutics recently released preclinical data on its gene upregulation technology, which selectively triggers gene expression within cells, as a treatment for spinal muscular atrophy (SMA). The data were given in a poster presentation at the recent Keystone Symposium for Chromatin and Epigenetics in British Columbia, Canada. SMA, a major cause of infant mortality, results from mutations or deletions of SMN1 gene…
