Research
Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
Dr. Chris Lorson and his colleagues have published a paper, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy,” in […]
Read More ›AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
AveXis recently released new data from the Phase 1 clinical trial of AVXS-101, a gene therapy program to treat SMA. The interim data through July […]
Read More ›2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. […]
Read More ›Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy
CK-2127107 is a novel fast skeletal muscle troponin activator being developed as a potential treatment for people living with SMA and certain other debilitating neuromuscular […]
Read More ›Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
Biogen and Ionis have been testing nusinersen in clinical trials and today announced that they will close ENDEAR, the Phase 3 trial testing nusinersen in […]
Read More ›Cure SMA Announces $2.5 Million in New Planned Research Funding
At the 2016 Annual SMA Conference, Cure SMA announced $2.5 million in new planned research funding over the next 12 months. This funding will be […]
Read More ›Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” […]
Read More ›Adelphi Values’ RO7350A Interview Study
A note from Cure SMA: We’re passing on information about this new interview study to the families in our community. It provides an opportunity to […]
Read More ›Cure SMA Awards $50,000 Drug Discovery Grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences
Cure SMA has awarded a $50,000 drug discovery grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences. The award is for […]
Read More ›Cure SMA Funds $2 Million in New Research in 2015-2016
Over the past few months, Cure SMA has announced a total of just over $2 million in new research funding. Our model is designed to […]
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