News

Trial of SMA Therapy RG7800 on Hold, but Roche, Partners Developing Another Compound

International pharmaceutical company Roche, with its SMA drug development collaboration partners PTC Therapeutics and the SMA Foundation, said a clinical study called Moonfish (NCT02240355) that was investigating a compound and investigational medicine known as RG7800 for people with spinal muscular atrophy (SMA) was placed on clinical hold in April 2015 after…

4 Areas of SMA Management

Approximately one in 10,000 newborns are born with spinal muscular atrophy (SMA), a life threatening condition that results in muscle wasting, severely impaired mobility, and sometimes a shortened life span in severe cases. Currently, SMA is incurable but there are ways to manage the condition and bring comfort to patients. Learn how…

New Report Recommends SMA Carrier and Perinatal Screening

Screening for spinal muscular atrophy (SMA) in high-risk groups could greatly reduce the impact of the disease, according to a new article published by researchers in Ottawa. The report, “Opening the window: The case for carrier and perinatal screening for spinal muscular atrophy,” was published in the journal Neuromuscular Disorders.

Promising Study of SMA Therapy in Mice Targets ‘Backup Gene’ SMN2

Cure SMA-funded researcher Dr. Christian L. Lorson and his colleagues have published a paper in the journal Molecular Therapy describing a promising therapeutic prospect in a mouse model with severe spinal muscular atrophy (SMA). The article, “Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal…

SMA Patients Being Recruited for Phase 2 Study of Skeletal Muscle Therapy

A Phase 2 clinical trial investigating a potential treatment, CK-2127107, in people with spinal muscular atrophy (SMA), ages 12 years and older, is currently recruiting patients in the U.S. with varying degrees of disease severity (Types 2, 3, or 4). CK-2127107 is an investigational drug candidate designed for skeletal muscle and other debilitating neuromuscular…