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Mashup Score: 1Loader - 7 day(s) ago
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Mashup Score: 0Loader - 7 day(s) ago
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Mashup Score: 1LDRTC Symposium: Newborn Screening and Lysosomal Disorders - 8 day(s) ago
This symposium, led by Ozlem Goker-Alpan, MD of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) reviews the current state of newborn screening (NBS) for lysosomal disorders and the need for newer guidelines to address the many unmet needs.
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Mashup Score: 2Rett Syndrome - 8 day(s) ago
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
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Mashup Score: 2Gene Therapy For Hemophilia B - 8 day(s) ago
Brahm Goldstein, MD, Vice President, R&D, Hematology at CSL Behring, discusses HEMGENIX, a pivotal gene therapy for hemophilia B.
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Mashup Score: 1Loader - 8 day(s) ago
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Mashup Score: 0Loader - 8 day(s) ago
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Mashup Score: 0Phelan-McDermid Syndrome and the JAG201 Study - 8 day(s) ago
Alex Kolevzon, MD, Clinical Director of the Seaver Autism Center at Mount Sinai, discusses Phelan-McDermid syndrome and the JAG201 study.
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Mashup Score: 1Multiple Myeloma and D-Rd vs. V-Rd Treatment - 8 day(s) ago
Doris Hansen, MD, discusses the treatment of multiple myeloma using D-Rd and V-Rd in transplant-ineligible patients.
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Mashup Score: 0Myelofibrosis Treatment Options - 8 day(s) ago
Hesham Abdullah, MD, MSc, of GSK, discusses myelofibrosis and the uses of momelotinib, also known as Ojjaara, to treat it.
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Andy Udell, President of Calliditas Therapeutics North America, discusses current IgA nephropathy treatment options (including Tarpeyo [budesonide]) and the Phase 3 NefIgArd trial #CheckRare #IgANephropathy https://t.co/TfRz7fX6qR