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Dr Edward Neilan of NORD discusses how the NORD Rare Disease Centers of Excellence has identified 11 potential working groups to tackle unmet needs...

Dr. John Jefferies discusses the importance of determining the etiology of LVH to determine if it is a genetic problem (such as Fabry...

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What Is AL Amyloidosis? - 6 days

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Dr. Jonathan Wall, Co-founder & Interim Chief Scientific Officer at Attralus, gives an overview of immunoglobulin light chain-associated (AL)...

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As part of the roundtable moderated by Philip Pearl, MD, of Boston Children’s Hospital and Harvard Medical School, the attendees of this virtual discussion...

Stefan M. Schieke, MD, Assistant Professor of Dermatology at University of Wisconsin-Madison, discusses the role of the dermatologist in diagnosing and treating cutaneous T-cell...

Modern treatment options for lysosomal storage disorders have created new...

Dr. PJ Brooks at NCATS describes the Platform Vector Gene Therapy (PaVe-GT) pilot...

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    P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences #NCATS describes the Platform Vector Gene Therapy (PaVe-GT) pilot project. @ncats_nih_gov @NIH https://t.co/zTvhQdO6Xu - view on twitter

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Dr. Caroline Hastings, of UCSF Benioff Children’s Hospital, discusses some the ‘tell tale’ signs of children with Niemann-Pick disease Type C...

Dr. Dalia Moawad, Head of Neurological Rare Diseases at Genentech, discusses 3-year data from the FIREFISH study testing risdiplam (Evrysdi) in infants with type...