Rare Disease Report


The pediatrician discusses key differences between treating pediatric and adult patients with hemophagocytic lymphohistiocytosis (HLH) with the recently FDA-approved emapalumab.  
Among the highlights observed in the data, the single-agent oral demonstrated significant improvement in progression-free survival (PFS).
Full results from the phase 3 MURANO trial showed a reduced risk in disease progression and 87.9% overall survival in relapsed/refractory chronic lymphocytic leukemia patients treated with venetoclax/rituximab.
Justin Taylor, MD, explains new and unique data that show cases in which a solid tumor can become a hematologic malignancy, or leukemia.
Males with myeloproliferative neoplasms (MPN) were found to have inferior survival compared to females in a well-defined, prospective, observational cohort.
The FDA has approved amifampridine (Firdapse) tablets as the first ever treatment of the rare autoimmune disorder, Lambert-Eaton myasthenic syndrome (LEMS) in adults.
The FDA has approved gilteritinib (XOSPATA) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.
Mayo Clinic investigators found young patients with myeloproliferative neoplasms who are under age 40 are a steadily growing population and seem to have longer survival than their older counterparts.
The FDA has granted an accelerated approval to larotrectinib (Vitrakvi, Bayer and Loxo Oncology, Inc) for the treatment of pediatric and adult solid tumors with neurotrophic receptor tyrosine kinase (NTRK) gene fusion.  
Here’s a round-up of recent designations granted by the FDA to products developed to treat rare diseases.
Copyright © RareDR 2013-2019 Rare Disease Communications. All Rights Reserved.