Author Archives: Ana de Barros PhD

Muscle Activators That Might Treat SMA at Forefront of Company’s 5-Year Plan

Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future — an initiative designed to expand the company’s drug pipeline and advance its muscle biology-directed drug candidates toward late-stage development and marketing strategies. The biopharmaceutical company specializes in first-in-class muscle activators for people with impaired muscle function, including spinal muscular atrophy (SMA) patients. The…

In Spinal Muscular Atrophy, Vascular System May Play a Key Role

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…

Cure SMA Publishes Clinical and Regulatory Webinar Q&A

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, was…

Cure SMA’s Medical Advisory Council Sets New Care Agenda

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 Given 10 New Feeder Seats By Jadon’s Hope Foundation

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 adaptive, comfortable and…

Genentech Updates Clinical Development of RG7800 for SMA

Genentech, a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions, recently announced an update on the clinical development of RG7800, its investigational SMN2 splicing modifier that is being developed for the treatment of patients with spinal muscular atrophy…

Low-Dose Irradiation to Edit The Genome of Patients’ Stem Cells

In a recent study published in the journal Stem Cells Translational Medicine, a team of researchers from the Cedars-Sinai Board of Governors Regenerative Medicine Institute have employed a gene-editing method that uses low-dose irradiation to edit the genome of patients’ stem cells. According to the study results, this strategy…

Cytokinetics Presents Positive Clinical Data For SMA Drug Candidate

During the SMA Annual Conference Cytokinetics presented results from their Phase 1 study of CK-2127107, a novel small molecule activator of the fast skeletal muscle troponin complex to improve skeletal muscle function in diseases and medical conditions associated with neuromuscular dysfunction, muscular weakness, and/or muscle fatigue. The company, in collaboration with Astellas, recently…