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Mashup Score: 1Intrauterine Treatment For Lysosomal Diseases - 16 hour(s) ago
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases.
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Mashup Score: 1Loader - 16 hour(s) ago
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Mashup Score: 1Seladelpar Beneficial for Patients With Primary Biliary Cholangitis - 16 hour(s) ago
Percentage of patients with biochemical response was higher in the seladelpar versus placebo group.
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Mashup Score: 0FDA Approves Casgevy to Treat Beta-Thalassemia - 22 hour(s) ago
Casgevy is the first CRISPR-based medicine to be approved for use in the United States for sickle cell disease and beta-thalassemia.
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Mashup Score: 0Enhanced Health Equity and Timely Diagnoses in Rare Diseases - 22 hour(s) ago
Cheryl Schwartz discusses Takeda’s report that calls for enhanced health equity and timely diagnosis for rare disease patients
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Mashup Score: 0
Triplet therapy of belantamab mafodotin, bortezomib, dexamethasone beneficial for relapsing or refractory multiple myeloma.
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Mashup Score: 3Myasthenia Gravis and Real-Life Data - 2 day(s) ago
Cynthia Qi, Health Economics Outcomes Researcher at Argenx, discusses myasthenia gravis and real-life data.
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Mashup Score: 0
Björn Mellgård, M.D., Ph.D., Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, breaks down Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
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Mashup Score: 3Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency - 2 day(s) ago
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young patients.
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Mashup Score: 0
No association seen for mAb during breastfeeding with infant hospitalization, systemic antibiotic use, developmental delay.
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Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases #CheckRare #LysosomalDisorders https://t.co/lxY2NBQcSj