Dublin — Mallinckrodt plc (NYSE American: MNK), a global specialty pharmaceutical company, announced that an abstract highlighting results from a retrospective analysis evaluating the corticosteroid-sparing effect and reduction of corticosteroid burden of Acthar® Gel (repository corticotropin injection) therapy in patients with advanced sarcoidosis, has been selected for a poster presentation...
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WUHAN, China and SAN DIEGO — Matthias Szabo Zarb, a Maltese college student, was diagnosed with Leber hereditary optic neuropathy caused by ND1 mutation (ND1-LHON) two years ago, which led to a rapid deterioration in his visual acuity and inability to engage in normal activities. Through the National Alliance for Rare...
WILMINGTON, Del. — Positive results from the MANDARA Phase III trial for FASENRA® (benralizumab) in patients with EGPA were published in the New England Journal of Medicine today, as the first head-to-head trial of biologics in patients with EGPA, and the first to demonstrate that more than half of patients...
THOUSAND OAKS, Calif. — Mandos Health® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that findings from analyses of its investigational therapy adrabetadex will be presented at the 54th Child Neurology Society (CNS) Annual Meeting, taking place on October 8-11, 2025 in Charlotte, North Carolina. Two analyses detail the clinical and...
LOS ANGELES – Mandos, LLC (Mandos) announced they have entered into an agreement with Vtesse, LLC, a wholly owned subsidiary of Mallinckrodt Pharmaceuticals (Mallinckrodt), a global biopharmaceutical company, to acquire adrabetadex (also known as VTS-270), a drug in development for the treatment of Niemann-Pick Type C1 disease (NPC1). This acquisition,...
DANBURY, Conn. and WESTLAKE VILLAGE, Calif. — MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with endocrine and orphan lung diseases, announced today that it has fully enrolled 305 patients living with type 1 or type 2 diabetes in its...
MANTECA — Manteca twins were born with a rare disease that affects just more than 1,200 globally. It’s left them stunted emotionally and physically. Augustin Polanco preps his twin 19-year-old daughters for school. Esperanza and Libertad, or Espy and Libby for short, suffer from a rare disease called syngap1. It’s...
SAN DIEGO, CA ― The targeted therapy combination of ibrutinib and venetoclax significantly improved progression-free survival (PFS) and achieved an overall remission rate in 82% of patients with relapsed/refractory mantle cell lymphoma (MCL), according to researchers at The University of Texas MD Anderson Cancer Center. Results from the Phase III...
PHOENIX, Arizona — High-grade gliomas are cancerous tumors that spread quickly in the brain or spinal cord. In a new study led by Mayo Clinic, researchers found invasive brain tumor margins of high-grade glioma (HGG) contain biologically distinct genetic and molecular alterations that point to aggressive behavior and disease recurrence. The findings...
Bunkyō, Japan – Thymic epithelial tumors are a rare group of malignancies originating in the thymus gland, that includes thymoma and thymic carcinoma. Among these, thymic carcinoma is the more aggressive subtype, characterized by high invasiveness, metastatic potential, and poor prognosis. With an incidence of just 0.15 cases per 100,000...