Alex Coletta,  —

Cure SMA has awrded a $50,000 Drug Discovery Grant to Brigham and Women’s Hospital researcher Kevin Hodgetts, PhD, at the Lab for Drug Discovery in Neurodegeneration for his project “Development of a Drug to Increase SMN2 Transcription.” The grant Dr. Kevin HodgettsPhoto Credit: Cure…

Cure SMA awarded a $50,000 drug discovery grant to researcher Barrington Burnett of Uniformed Services University of the Health Sciences for the project “Slowing SMN degradation to treat SMA,” which aims to characterize and validate a new survival motor neuron (SMN) protein modulator for the treatment of spinal muscular atrophy (SMA). Drug discovery…

Cure SMA has just released an update on its spinal muscular atrophy (SMA) drug pipeline and, according to a press release, the latest version now includes increased coverage of potential treatment approaches to SMA: 18 active programs, 14 pharmaceutical partners, six programs in clinical trials, and 28 programs…

Biogen and Ionis Pharmaceuticals have recently issued several updates on the clinical development of nusinersen, an antisense drug designed to treat spinal muscular atrophy (SMA). The partners announced that the enrollment period is now completed for both Phase 3 trials and for the Phase 2 study. Phase 3 trials remain on…

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 spinal…

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.

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…