Showing results (11-20 of 126) with videos related to
Sort By:
Pageof 13
Therapeutic Advances in Rare Disease|May 14, 2023
Wiskott-Aldrich syndrome with normal platelet volume in a low-income setting: a case reportWilliam Frank Mawalla, Hamisa Iddy, Christine Aloyce Kindole, et al.Therapeutic Advances in Rare Disease|September 15, 2025
Systemic inflammation in Fabry disease: a longitudinal immuno-genetic analysis based on variant stratificationHaylen Marín Gómez, Miguel López-GarridoTherapeutic Advances in Rare Disease|July 14, 2025
Enhancing and leveraging principal investigator and patient advocacy group collaboration in rare disease clinical research-meeting report from the rare Diseases Clinical Research NetworkKristen Wheeden, Sheridan Meyers, Kristin Anthony, et al.Therapeutic Advances in Rare Disease|December 18, 2024
Advancing rare disease measurement through the Rare Disease Clinical Outcome Assessment ConsortiumNaomi Knoble, Lindsey T MurrayTherapeutic Advances in Rare Disease|July 31, 2024
Koolen-de Vries Syndrome: a journey from diagnosis to treatmentsAnna C Pfalzer, Blake Ivers, Alayna Haynam, et al.Therapeutic Advances in Rare Disease|June 24, 2024
Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trialsJayEtta Hecker, Gabrielle Conecker, Chere Chapman, et al.Therapeutic Advances in Rare Disease|August 17, 2023
Enhancing diversity, equity, inclusion, and accessibility in eosinophilic gastrointestinal disease research: the consortium for eosinophilic gastrointestinal disease researchers' journeyMirna Chehade, Glenn Furuta, Amy Klion, et al.Therapeutic Advances in Rare Disease|December 20, 2023
The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry PlatformJanet Sultana, Nikita Camilleri, Salvatore Crisafulli, et al.Therapeutic Advances in Rare Disease|May 8, 2024
Establishing resources and increasing awareness to advance research on Dentatorubral-pallidoluysian atrophy toward a treatment: a patient organization perspectiveSilvia Prades, Andrea Compton, Jeffrey B CarrollTherapeutic Advances in Rare Disease|September 27, 2024
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA TherapeuticsAnnemieke Aartsma-Rus, Rob W J Collin, Ype Elgersma, et al.Pageof 13