Challenges Facing Rare Disease Clinical Trials | CheckRare.com
Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions.
Nick Kenny, PhD, Chief Scientific Officer at Syneos Health talks about the challenges in developing clinical trials for rare conditions.
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
Stiff person syndrome (SPS) is a rare, progressive disease that affects the nervous system, specifically the brain and spinal cord.
Pisit “Duke” Pitukcheewanont, MD, Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 for treatment of growth hormone deficiency.
Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments.
Jula Inrig, MD, discusses a clinical trial that tested sparsentan for treatment of IgA nephropathy, compared to traditional RAS inhibitors.
Kurt Gunter, MD, Inozyme Pharma, discusses data presented on ENPP1 deficiency and ABCC6 deficiency at ASBMR 2024.
Bruce Cree, MD, PhD, discusses a post hoc analysis of patient pain and quality of life data in treatment with Uplizna for NMO.
Andra Stratton, President and Co-founder of Lipodystrophy United, discusses the challenges of lipodystrophy from a patient’s perspective.
Lisa Berry, Genetic Counselor at Cincinnati Children’s Hospital, discusses results from Fabry patient survey.
Paul Bolno and Anne-Marie Li-Kwai-Cheung of Wave Life Sciences, discuss interim data from the FORWARD-53 trial for DMD.