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  • Neurology
  • James P. Van Meerbeke, Research Assistant from the lab of Charlotte J. Sumner, M.D., Associate Professor of Neurology and Neuroscience, Johns Hopkins University School of Medicine, will present the abstract titled " The Therapeutics Effects of RG3039 in Severe Spinal Muscular Atrophy - Mice and Normal Human Volunteers ," during "The Future of Neuroscience Conference: Neurologists and Neuroscientists Defining the Next Generation of CNS Therapies," taking place on April 27. (thestreet.com)
  • Cytokinetics, Incorporated (Nasdaq:CYTK) today announced data from the Phase 2 clinical study of reldesemtiv in patients with spinal muscular atrophy (SMA) were presented in an oral presentation by John W. Day, M.D., Ph.D., Professor of Neurology and Pediatrics (Genetics), Stanford University, at the 2018 Annual Cure SMA Conference in Dallas. (advfn.com)
  • There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life," said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. (wordpress.com)
  • infants
  • In the study of 20 babies aged one to seven months, researchers tested the safety, effectiveness and mechanism of action of the nusinersen drug, which is designed to modify the way the body reads the SMN2 gene to increase the production of SMN protein in these infants and children with spinal muscular atrophy. (eurekalert.org)
  • severity
  • Analyte associations were evaluated against a primary measure of disease severity, the Modified Hammersmith Functional Motor Scale (MHFMS) and to a number of secondary clinical measures. (plos.org)
  • 2016
  • This new investigational medicine will now advance to its very first clinical study in healthy individuals in January 2016, which will provide important information about the safety profile, pharmacokinetics (what the body does to the medicine) and effects of RG7916 in healthy individuals. (sma-europe.eu)
  • Roche
  • The initiation of the clinical development triggers a $7.5 million payment to PTC from Roche. (ptcbio.com)
  • Genentech, a member of the Roche Group, announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). (medicalnewser.com)
  • Roche and Genentech are leading the clinical development of risdiplam, an oral SMN2 splicing modifier, as part of a collaboration with the SMA Foundation and PTC Therapeutics. (medicalnewser.com)
  • drug
  • RG3039 is the first clinical-stage drug candidate to target the core genetic deficit in SMA in order to treat the biochemical deficits caused by decreased levels of the survival motor neuron (SMN) protein. (thestreet.com)
  • The drug was injected directly into the spinal fluid via a lumbar puncture. (eurekalert.org)
  • Our report exclusively focuses on providing insights and competitive opportunities based on Biomarker or target receptor, Drug Class and Mechanism of Action to aid in understanding the upcoming newer research trends in the field of Spinal Muscular Atrophy drug discovery. (gervanora.com)
  • 7 million milestone payment from Biogen Idec , to advance an ongoing open-label extension clinical study of the drug ISIS-SMNRx in children with spinal muscular atrophy (SMA). (smanewstoday.com)
  • Foundation
  • This study was sponsored by the Spinal Muscular Atrophy (SMA) Foundation, New York, New York, who also had a role in the design of the study, data collection and analysis, decision to publish, and preparation of the manuscript ( http://www.smafoundation.org/ ). (plos.org)
  • studies
  • In these preclinical studies, which provided proof of principle for conducting human clinical studies, administration of RG3039 resulted in a significant improvement in survival, increased maximum body weight and improved motor behavior in severe SMA mice. (thestreet.com)
  • development
  • Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA), a patient advocacy organization that funded and directed the preclinical development of RG3039 with an investment of more than $13 million. (thestreet.com)