Francesca Carubbi, MD, PhD, of the University of Modena and Reggio Emilia, Italy discusses the impact the COVID-19 pandemic has had on lysosomal storage and rare disorders in Italy.
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.
Monica Gadelha, DR, PhD, at the University of Rio De Janeiro discusses the PATHFNDR-1 trial and acromegaly.
Dr. La Piana gives advice to patients and caregivers on diagnosing adult onset leukoencephalopathy with axonal spheroids and pigmented glia.
Approval allows Onivyde (plus oxaliplatin, fluorouracil, and leucovorin) as first-line therapy.
Tyler Sandahl, MD, Clinical Pharmacist at the Mayo Clinic, discusses the use of Tocilizumab in multiple myeloma patients.
Cat Lutz, Ph.D., M.B.A. discusses how Jackson Laboratory uses mice in testing for human disease.Cat Lutz, Ph.D., M.B.A. Vice President of the Rare Disease Translational…
