Johns Hopkins University researcher Dr. Charlotte Sumner, M.D., has been granted a Cure SMA award of $140,000 for her project, “Assessing the reversibility of proximal axon abnormalities in SMA mice.” Photo Credit: Cure SMA Sumner is a specialized adult neurologist with training in neuromuscular disease and…
Astellas Pharmaceuticals is featuring Cure SMA on its new ‘Changing Tomorrow Together’ website, an online resource created by the company to stimulate dialogue and the sharing of ideas and information across disease advocacy communities. The Cure SMA feature is a “spotlight piece” written by the group’s research director, Jill Jarecki.
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…
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…
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…
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,…
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…
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…
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…
