Therapeutic Potential of IGF-1 and MECP2 in Rett Syndrome
April 16th 2023Research that is already underway will be necessary to confirm the safety and efficacy of this class of Rett syndrome therapies, and targeting IGF-1 may be a possibility for treating additional neurological disorders beyond Rett.
Therapeutics Aim at Modifying Alzheimer Disease
April 16th 2023Disease-modifying agents will require early detection, including comprehensive evaluation of disease severity and alternative or confounding diagnoses. When the long-awaited moment arrives, when disease-modifying therapies finally are available for ADRDs…will we be ready?
Evolution of High Intensity Focused Ultrasound to Treat Essential Tremor: Justin Sporrer, MD
April 14th 2023The director of functional neurosurgery at Baptist Health provided perspective on the advances of high intensity focused ultrasound as the institution nears 100 patients treated. [WATCH TIME: 4 minutes]
Clinical Application of Induced Pluripotent Stem Cells in Muscular Dystrophy: Rita Perlingeiro, PhD
April 13th 2023The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2023 conference. [WATCH TIME: 5 minutes]
NeuroVoices: Julie Pilitsis, MD, PhD, MBA, on Weighing Surgical Options for Degenerative Conditions
April 12th 2023The neurosurgeon at Marcus Neuroscience Institute, Baptist Health, provided perspective on invasive procedures for degenerative conditions and when deep brain stimulation is needed.
Assessing New Long-Term Efficacy and Durability Data of Zolgensma for Spinal Muscular Atrophy
April 11th 2023Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference.
Predicting Clinical Needs and Potential for Genetic Therapies in ALS: Jennifer Morganroth, MD, MBA
April 11th 2023At the 2023 MDA’s Clinical and Scientific conference, the neurology resident at Penn Medicine talked about the challenges of delivering intrathecal therapies and the importance of planning clinical resources for patients with ALS. [WATCH TIME: 3 minutes]