Ionis Pharmaceuticals announced it has completed its target enrollment of the Phase 3 CHERISH study, a trial designed to support marketing approval of the drug candidate nusinersen in children with spinal muscular atrophy (SMA). The achievement earned Carlsbad, California-based Ionis a milestone payment of $2.15 million from Biogen, of Cambridge, Mass. According…
Roche recently announced that a new investigational medicine for the treatment of spinal muscular atrophy (SMA) — RG7916 — will soon advance to its first clinical trial. The study will provide information on the safety and tolerability, and investigate the pharmacokinetics (absorption, distribution, metabolism and excretion) of RG7916 in healthy individuals. In April 2015, the Moonfish clinical…
Ionic Pharmaceuticals announced a landmark payment of $5 million from Biogen for the validation of an unnamed target to treat patients with an as-yet unrevealed neurological disorder. Ionis and Biogen have an extensive strategic partnership to develop drugs for neurological disorders; Ionis will continue to evaluate the newest target to…
Researchers have developed a novel method for detecting carriers of spinal muscular atrophy (SMA) using next generation sequencing screening. The new technique can easily be incorporated into current next generation sequencing screening panels, analyzing hundreds of genes involved in severe childhood diseases. While a rare disease, SMA is still the…
Cure SMA recently published a 2015 review of its advocacy work, highlighting the programs it provides to families living with Spinal Muscular Atrophy (SMA) that aid in caring for those with SMA and offering the emotional support of a unified community who understands the struggles of the disease. In its efforts to support the continued…
Cure SMA of Elk Grove Village, Illinois, and biopharmaceutical company Cytokinetics Inc. of San Francisco, California, announced an expanded partnership to increase public awareness, education, and fund-raising efforts for spinal muscular atrophy (SMA). As a Cure SMA National Gold Partner, Cytokinetics will lend support to key national and local initiatives…
For the first time, scientists have shown that insufficient blood supply is likely to contribute to motor neuron loss in spinal muscular atrophy (SMA), a finding that potentially opens a new avenue into disease research. The study, entitled “Vascular defects and spinal cord hypoxia in spinal muscular atrophy,” was…
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…
