The degeneration of spinal motor neurons in spinal muscular atrophy (SMA) may be the direct result of abnormal mitochondrial (energy source of cells) transport and morphology and subsequent reduced mitochondrial health. These are the conclusions of a study preformed by an international team of collaborative researchers led by scientists from the…
The non-profit organization Cure SMA has published the video and transcription of an event recently organized, titled “Clinical Regulatory Webinar.” On Monday, November 9th, Cure SMA hosted a one-hour webinar to discuss clinical trial design, expanded access, New Drug Application (NDA) processes, among other topics. Jill Jarecki, the research director at Cure SMA,…
U.K. researchers funded by the SMA Trust have found that insufficient blood supply may well contribute to motor neuron loss in spinal muscular atrophy (SMA). The study entitled, “Vascular defects and spinal cord hypoxia in spinal muscular atrophy,” was published in the latest edition of the Annals of…
Members of Cure SMA‘s Medical Advisory Council (MAC) recently met for two days in Chicago to set the agenda for the care of SMA individuals and families. Mary Schroth, MD, a leading pulmonologist and professor of pediatrics, chairs the MAC, one of the most respected groups of SMA medical and clinical specialists in…
Cure SMA has been given 10 special tomato feeder seats by the Jadon’s Hope Foundation. The donation is meant to help the organization in its goal of not only finding a cure for spinal muscular atrophy (SMA) but also improving the quality of life for patients with this severe condition. The…
Cytokinetics has announced additional details about a planned Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA). Investigators believe CK-2127107, a skeletal muscle activator, has the potential to improve muscle function, either alone or in combination with other drugs. The company, in collaboration with its partner Astellas, recently held…
Scientists granted initial funding worth $50,000 from Cure SMA recently published a study that found obese children suffering from SMA Type 2 had a higher risk of developing impaired glucose tolerance, regardless of their degree of visual obesity. The lead investigators of the study, “Responses to Fasting and Glucose Loading…
Isis Pharmaceuticals, Inc. recently announced the official launch of an open-label extension study, called SHINE, which will be continuing treatment of the same infants and children diagnosed with spinal muscular atrophy (SMA) who had previously participated in Phase III studies ENDEAR and CHERISH. The infants whi had participated in ENDEAR will continue to…
Researchers from The Jackson Laboratory in Maine, in collaboration with Isis Pharmaceuticals, tested a novel therapeutic candidate (antisense oligonucleotides) in genetically engineered mice with spinal muscular atrophy (SMA), observing it could restore some functionality of motor neurons. The study entitled “Systemic, postsymptomatic antisense oligonucleotide rescues motor…
AveXis, Inc. has recently presented results from its ongoing Phase 1/2 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1 during the International Congress of the World Muscle Society in Brighton, United Kingdom. Lead investigator Dr. Jerry R. Mendell, director of the Center for Gene…
