Panel Discussion: The High Cost of Rare Diseases
Dr. Joni Rutter of NCATS and Annie Kennedy of the EveryLife Foundation discuss four recent studies estimating the direct and indirect costs of rare diseases in the United States.
Related Articles
Triplet Therapy Including Belamaf Prolongs Survival in Multiple Myeloma
Triplet therapy of belantamab mafodotin, bortezomib, dexamethasone beneficial for relapsing or refractory multiple myeloma.
Myasthenia Gravis and Real-Life Data
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.
PATHFNDR 1 Trial and Acromegaly
Monica Gadelha, DR, PhD, at the University of Rio De Janeiro discusses the PATHFNDR-1 trial and acromegaly.
Diagnosing Adult-Onset Leukoencephalopathy
Dr. La Piana gives advice to patients and caregivers on diagnosing adult onset leukoencephalopathy with axonal spheroids and pigmented glia.
FDA Approves Onivyde Combination for Metastatic Pancreatic Cancer
Approval allows Onivyde (plus oxaliplatin, fluorouracil, and leucovorin) as first-line therapy.
Tocilizumab in Multiple Myeloma Patients
Tyler Sandahl, MD, Clinical Pharmacist at the Mayo Clinic, discusses the use of Tocilizumab in multiple myeloma patients.
JAX Labratory and the Use of Mice in Rare Genetic Disease Testing
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…