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Percentage of patients with biochemical response was higher in the seladelpar versus placebo group.
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Casgevy is the first CRISPR-based medicine to be approved for use in the United States for sickle cell disease and beta-thalassemia.
Enhanced Health Equity and Timely Diagnoses in Rare Diseases
Cheryl Schwartz discusses Takeda’s report that calls for enhanced health equity and timely diagnosis for rare disease patients
Triplet Therapy Including Belamaf Prolongs Survival in Multiple Myeloma
Triplet therapy of belantamab mafodotin, bortezomib, dexamethasone beneficial for relapsing or refractory multiple myeloma.
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Cynthia Qi, Health Economics Outcomes Researcher at Argenx, discusses myasthenia gravis and real-life data.
Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Overview
Björn Mellgård, M.D., Ph.D., Vice President, Global Program Lead, Rare Genetics and Hematology, Takeda, breaks down Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young patients.
AAN: Monoclonal Antibodies for MS During Breastfeeding Not Harmful
No association seen for mAb during breastfeeding with infant hospitalization, systemic antibiotic use, developmental delay.