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Therapeutic Advances in Rare Disease|May 14, 2023
Utilizing telehealth to create a clinical model of care for patients with Batten disease and other rare diseasesJessica F Scherr, Charles Albright, Emily de Los ReyesTherapeutic Advances in Rare Disease|May 29, 2024
Five cases of pulmonary Aspergillus nodules diagnosed at surgery and by pathology in immunocompetent patients, with a literature reviewShuangxia Dong, Fengxiang Wang, Haizhen Jin, et al.Therapeutic Advances in Rare Disease|August 25, 2023
Toward representative genomic research: the children's rare disease cohorts experienceZoë J Frazier, Eurnestine Brown, Shira Rockowitz, et al.Therapeutic Advances in Rare Disease|May 1, 2023
Impact of the Covid-19 epidemic on a US sample of patients with myasthenia gravisGloria Gutierrez, Helen Girma, Pierce Kuhnell, et al.Therapeutic Advances in Rare Disease|December 29, 2025
From care to cure: a patient engagement framework for rare disease and orphan drug researchNahya Awada, Anil VarugheseTherapeutic Advances in Rare Disease|December 30, 2025
Lysosomal storage diseases in North America: a comprehensive review of enzyme therapies and unmet needsLunawati L BennettTherapeutic Advances in Rare Disease|July 14, 2025
Finding buried genetic test results in the electronic health record is inefficient and variable across institutionsOlivia J Veatch, Jomol Mathew, Shira Rockowitz, et al.Therapeutic Advances in Rare Disease|September 22, 2025
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO studyPerry B Shieh, Wendy Hughes, Marie Wood, et al.Therapeutic Advances in Rare Disease|July 22, 2025
Sparsentan in IgA nephropathy: a plain language summary of publication for the PROTECT studyBrad H Rovin, Radko Komers, Chris Scroggins, et al.Therapeutic Advances in Rare Disease|August 4, 2025
Updated demographics categories to capture the true diversity of an international registry of rare disease patientsNicole Kressin, Michael E Shy, Tara Jones, et al.Pageof 13