ALS frontiers: accelerating diagnosis and access in rare disease

Annie Kennedy is the Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases and has led major initiatives to improve rare disease care including the National Economic Burden of Rare Disease study, the Cost of Delayed Diagnosis project, and the development of the ICD Code Roadmap. Annie serves on numerous advisory boards and steering committees, including the NIH NCATS Advisory Council, the Cures Accelerator Network Advisory Board, and the Patient Driven Values in Healthcare Evaluation Steering Committee.

Nothing to Disclose.

Laynie Dratch is a licensed, certified genetic counselor at the University of Pennsylvania Perelman School of Medicine. She received her ScM in genetic counseling from the Johns Hopkins University / National Institutes of Health training program. She specializes in the care and research of frontotemporal degeneration and ALS spectrum disorders.

Employee of Penn Medicine. Current or former consulting/advisory relationships with Sano Genetics, Passage Bio, and Biogen.

Suma Babu is an Associate Professor of Neurology at Harvard Medical School and co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital. Her work specializes in the care and research of ALS and motor neuron diseases. She leads neuroimaging research programs focused on disease biomarkers and therapeutic development, and has served as a principal investigator for multiple Phase 1–3 ALS clinical trials.

Grants from NIH/NINDS; research funding from Ionis, Biogen, Novartis, OrphAI, and Denali; consulting fees to institution from uniQure and MarvelBiome; and honoraria from NIH and American Academy of Neurology

Terry Heiman-Patterson is a Professor of Neurology at the Lewis Katz School of Medicine at Temple University and Director of the Temple MDA/ALS Center of Hope. In her work focusing on ALS and related motor neuron diseases, she has led multidisciplinary clinical programs and research focused on the mechanisms of motor neuron disease, therapeutic development, and patient-centered care. She has served as Co-Chair of the Northeast ALS Consortium Executive Committee and is a member of the ALS Research Group.

Funding from the Muscular Dystrophy Association, a clinical trial readiness grant from the ALS Association, Independent Investigator Funding from MTPA, medical advisory boards at MTPA, Amylyx, Cytokinetics, and Biogen, and clinical trial funding from Amylyx, Healey Trial, Cytokinetics, PKC, and MTPA.