Videos

Thoughts on Clinical Trials from the Perspectives of an ALS Center Director
01:12:45

Thoughts on Clinical Trials from the Perspectives of an ALS Center Director

Register to upcoming events everythingals.org/events Participate in our ALS Research everythingals.org/research Thoughts on Clinical Trials: From the Perspectives of an ALS Clinical Director Our Guest Speaker, Jonathan S. Katz, M.D., is a neurologist in San Francisco, California. Dr. Katz is affiliated with multiple hospitals in the area, including Sutter Medical Center and California Pacific Medical Center. Dr. Katz studies ALS, myasthenia gravis, neuromuscular diseases, pain management, peripheral neuropathy, poliomyelitis, radiculopathies, and spinal muscular atrophy. He leads clinical trials of pharmacotherapies to slow the progression of ALS; device trials; a study of the NeuRX Diaphragm Pacing System to support patients with diaphragm weakness; and observational studies of factors affecting disease progression. Dr. Katz and others at the Forbes Norris Center work with researchers at the University of California, San Diego, to study mutations in RNA binding proteins that cause neuronal dysfunction and death. In 2014, Dr. Katz and colleagues at CPMC will collaborate with Cedars-Sinai Regenerative Medicine Institute on new stem cell and gene therapy techniques to treat ALS patients. He is President of the ALS Research Group—an international ALS research collaboration. He sits on the medical advisory board for GBS-CIDP Foundation International and the Myasthenia Gravis Association. DO NOT Miss Another Event, Subscribe to our Channel: youtube.com/c/everythingals
Expanded Access for Rare Diseases
01:00:08

Expanded Access for Rare Diseases

Register to upcoming events everythingals.org/events Participate in our ALS Research everythingals.org/research Functional Medicine and Microbiome Alison​ ​Bateman​-​House​, ​PhD​, ​MPH, MA​ ​is​ ​an​ ​Assistant​ ​Professor​ ​in​ ​the​ ​Division​ ​of​ ​Medical Ethics​ ​at​ ​NYU​ ​Grossman​ ​School​ ​of​ ​Medicine​ and a Professor of Practice in Law at University of Arizona James E. Rogers College of Law. ​She​ ​is​ ​co​-​chair, with Arthur Caplan PhD,​ ​of​ ​the​ ​Working​ ​Group​ ​on Compassionate​ ​Use​ ​and​ ​Preapproval​ ​Access​ (​CUPA​), ​an​ ​academic group​ ​that​ ​studies​ ​ethical issues concerning access​ ​to​ ​investigational​ ​medical products​ ​and which consists of patient advocates, clinicians, members of industry, current and former FDA staffers, lawyers, and academics, among others. Dr Bateman-House also co-chairs, with Lesha Shah MD, the Pediatric Gene Therapy and Medical Ethics (PGTME) working group, which includes academics, patient advocates, biopharmaceutical industry representatives, and a wide array of clinical and research professionals. Dr. Bateman-House advises a wide array of biopharmaceutical companies, patient advocacy organizations, governmental and non-governmental entities about clinical trial design and non-trial access programs, and she serves as ethicist for multiple data safety monitoring boards overseeing clinical trials. Dr. Bateman​-​House​ also serves​ ​as​ ​the non-voting chair​ ​of​ ​the​ ​NYU​/​Janssen Pharmaceutical Compassionate​ ​Use​ ​Advisory​ ​Committees​ (​CompACs) for​ ​Infectious​ ​Diseases and​ ​Neurology/Psychology​. CompAC won the Reagan-Udall Foundation for the FDA’s 2019 Innovation Award. She has published and spoken extensively on non-trial access to investigational drugs, ​on​ ​clinical​ ​trial​ ​accessibility​, on​ ​the​ ​history​ ​and ethics​ ​of​ ​using​ ​humans​ ​as​ ​research​ ​subjects​, and on public health ethics. DO NOT Miss Another Event, Subscribe to our Channel: youtube.com/c/everythingals
2022 Outlook for ALS
01:40:04

2022 Outlook for ALS

Register to upcoming events everythingals.org/events Participate in our ALS Research everythingals.org/research 2022 Outlook for ALS Jinsy A. Andrews, M.D., M.Sc., FAAN is currently an Associate Professor of Neurology, in the Division of Neuromuscular Medicine and serves as the Director of Neuromuscular Clinical Trials at Columbia University. She currently oversees neuromuscular clinical trials and cares for patients with neuromuscular disease, primarily ALS. Dr. Andrews has extensive experience in all phases of human clinical trials and drug development in both the academic and industry settings. Dr. Andrews is the elected co-chair of the Northeastern ALS (NEALS) Consortium, and also elected to the National Board of Trustees of the ALS Association and is a Fellow of the American Academy of Neurology (FAAN). Dr. Andrews has also received the Diamond Award for ALS clinical research from Wings Over Wall Street and the Muscular Dystrophy Association. Dr. Andrews gives us an amazing overview on the 2022 Outlook for ALS, including discussions on current FDA approved treatments for ALS, how to get access to investigational drugs, treatments in the pipeline, and multidisciplinary care for ALS management including nutritional and respiratory care. Dr. Andrews also dives into clinical trials: the pitfalls, errors, and how to choose the proper trial for you. She also discusses Expanded Access and many of the questions regarding ACT for ALS and what this means for getting access to investigational drugs. All this and much more! DO NOT Miss Another Event, Subscribe to our Channel: youtube.com/c/everythingals