Patient Advocacy, Pandemic Impact | CheckRare
Amy Blum of the National Gaucher Foundation discusses the important role patient advocacy groups can play in helping the rare disease community during the current COVID-19 pandemic.
Amy Blum of the National Gaucher Foundation discusses the important role patient advocacy groups can play in helping the rare disease community during the current COVID-19 pandemic.
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Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases.
Project FIND-OUT enables the rapid detection of rare genetic neurodevelopmental disorders in babies ages 3-12 months.
Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett…
Monica Gadelha, DR, PhD, University of Rio De Janeiro, discusses her acromegaly research.
Davide Martino, discusses the BASIS clinical trial, a phase 3 study testing marstacimab in patients with hemophilia without inhibitors.
Monica Gadelha, DR, PhD, at the University of Rio De Janeiro discusses the PATHFNDR-1 trial and acromegaly.
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Tyler Sandahl, MD, Clinical Pharmacist at the Mayo Clinic, discusses the use of Tocilizumab in multiple myeloma patients.
Triplet therapy of belantamab mafodotin, bortezomib, dexamethasone beneficial for relapsing or refractory multiple myeloma.