Alexandra Alizadeh is the CEO of Adviise, a medical technology firm dedicated to connecting patients to specialist clinicians. In light of the recent COVID-19 pandemic, she notes an increase in telehealth among most health care providers, something she sees as a positive thing.
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases.
Percentage of patients with biochemical response was higher in the seladelpar versus placebo group.
Casgevy is the first CRISPR-based medicine to be approved for use in the United States for sickle cell disease and beta-thalassemia.
Cheryl Schwartz discusses Takeda’s report that calls for enhanced health equity and timely diagnosis for rare disease patients
Triplet therapy of belantamab mafodotin, bortezomib, dexamethasone beneficial for relapsing or refractory multiple myeloma.
Cynthia Qi, Health Economics Outcomes Researcher at Argenx, discusses myasthenia gravis and real-life data.
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 is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young patients.
No association seen for mAb during breastfeeding with infant hospitalization, systemic antibiotic use, developmental delay.
