Access to Treatment Committee

The Access to Treatment Committee reviews proposals from physicians or organizations, like the UDN, to treat diagnosed, genetically confirmed ultra-rare disease patients. Proposals will be approved and prioritized based on criteria such as: feasibility of developing an ASO treatment for the genetic cause of the disease, degree of potential benefit of treatment, practicality of treatment, availability of physician and institution to treat patient and conduct necessary studies, and other complexities of the condition.

At the Foundation we understand that there are seriously ill patients with immediate needs of a treatment for their condition. We are sympathetic to patients and all who are involved. The Committee will fully review all proposals received before a decision is made. We plan to do all we can to make sure we are as transparent as possible. Due to the complex nature of treating patients with ultra-rare diseases, unfortunately not all can be approved for treatment.

Learn more about the committee.

Our Access to Treatment Committee is made up of pharmaceutical executives, physicians, academic professors, patient advocates, and a bioethicist. This committee will review proposals from physicians or organizations, like the UDN, to treat diagnosed, genetically confirmed ultra-rare disease patients.

  • Frank Bennett, PhD, Co-ChairmanChief Technology Officer at n-Lorem and Chief Scientific Officer at Ionis Pharmaceuticals
  • Joseph G. Gleeson, MDChief Medical Officer at n-Lorem and Rady Professor, Neurosciences and Pediatrics, University of California, San Diego
  • Alan Beggs, PhD – Professor of Pediatrics at Boston Children’s Hospital
  • Lauren Black, PhDDistinguished Scientist at Charles River
  • Kristina BowyerPatient Advocate at Ionis Pharmaceuticals
  • Jeff Carroll, PhDAssistant Professor in the Behavioral Neuroscience Program and the Department of Psychology at Western Washington University
  • Francis Sessions Cole, III, MDProfessor of Pediatrics at Washington University School of Medicine in St. Louis
  • Darryl De Vivo, MDSidney Carter Professor of Neurology, Professor of Pediatrics, and Director Emeritus (1979-2000) of the Child Neurology Service at Columbia University Irving Medical Center, New York City
  • Richard Finkel, MDProfessor of Neurology at the University of Central Florida School of Medicine, and Chief, Division of Neurology at Nemours Children’s Hospital in Orlando
  • Kenneth ‘Kurt’ Fischbeck, MDNIH Distinguished Investigator, Neurogenetics Branch
  • Willis Maddrey, MDProfessor of Internal Medicine and Assistant to the President, University of Texas, Southwestern Medical Center at Dallas
  • Matthew Might, PhDDirector at Hugh Kaul Precision Medicine Institute at University of Alabama at Birmingham
  • Jeffrey Noebels, MD, PhDCullen Chair in Neurogenetics and Professor of Neurology, Neuroscience, and Molecular and Human Genetics at Baylor College of Medicine
  • Alfred Sandrock, Jr, MD, PhDExecutive Vice President, Research & Development and Chief Medical Officer at Biogen
  • Eugene Schneider, MD, PhDVice President and Head of Clinical Development at Ionis Pharmaceuticals
  • Neil Shneider, MD, PhDAssociate Professor of Neurology in the Neuromuscular Division at Columbia University,  Vagelos College of Physicians and Surgeons
  • Eric Swayze, PhDVice President Research at Ionis Pharmaceuticals
  • Timothy Yu, MD, PhDAttending Physician, Division of Genetics and Genomics & Assistant Professor in Pediatrics at Harvard Medical School