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…
Children with spinal muscular atrophy (SMA) have weaker muscles than children who are not affected by muscle myopathies and new results from a study conducted at Washington University School of Medicine in St. Louis, Missouri, indicate this muscle weakness may be due to differences in muscle thickness. Researchers used ultrasound…
The Ensuring Access to Clinical Trials Act of 2015 (EACT) has been passed by the U.S. House of Representatives, and having already been passed the Senate in July, the bill will now go to President Obama to be signed into law. The EACT bill authorizes individuals to receive up to…
Cure SMA, an organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently revealed novel studies presented during its annual researcher meeting. SMA is a genetically-determined neuromuscular disorder characterized by loss of specific spinal cord neurons, which causes weakness of the limbs and muscle degeneration. This leads to loss of voluntary muscle…
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