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Therapeutic Advances in Rare Disease|March 17, 2025
Frequent report of vitamin deficiencies and use of supplements and complementary/alternative treatment approaches in patients with eosinophilic gastrointestinal diseasesBrenderia A Cameron, Elizabeth T Jensen, Xiangfeng Dai, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?Luiza Amara Maciel Braga, Carlos Gilbert Conte Filho, Fabio Batista MotaTherapeutic Advances in Rare Disease|May 14, 2023
Building cross-border collaborations to increase diversity and accelerate rare disease drug development - meeting report from the inaugural IndoUSrare Annual Conference 2021Harvinder Kour Khera, Nisha Venugopal, Ramya T Karur, et al.Therapeutic Advances in Rare Disease|May 14, 2023
End-stage crystalline maculopathy with retinal atrophy in Sjögren-Larsson syndrome: a case report and review of the literatureLester H Lambert, Noreen Shaikh, Jeffrey L Marx, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Congenital disorder of glycosylation - one size does not fit all: a parent's perspectiveKonstantin FeinbergTherapeutic Advances in Rare Disease|May 14, 2023
Successful combined umbilical cord blood and bone marrow transplantation from an HLA-matched sibling for MPS VI: a case reportPankti Haria, Vinayak Kedage, Pradnya Dalvi, et al.Therapeutic Advances in Rare Disease|May 14, 2023
They've been BITTEN: reports of institutional and provider betrayal and links with Ehlers-Danlos Syndrome patients' current symptoms, unmet needs and healthcare expectationsJennifer Langhinrichsen-Rohling, Chrystal L Lewis, Sean McCabe, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Etranacogene dezaparvovec for hemophilia B gene therapyCourtney D ThornburgTherapeutic Advances in Rare Disease|May 14, 2023
Beyond pigmentation: signs of liver protection during afamelanotide treatment in Swiss patients with erythropoietic protoporphyria, an observational studyAnna-Elisabeth Minder, Jasmin Barman-Aksoezen, Mathias Schmid, et al.Therapeutic Advances in Rare Disease|May 14, 2023
Enzyme replacement therapy in lysosomal acid lipase deficiency (LAL-D): a systematic literature reviewAamir Bashir, Pramil Tiwari, Ajay DusejaPageof 13