Tag: disease progression

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®.

PARIS, FRANCE, 24 July 2025 - Ipsen announced today that the European Commission has approved Cabometyx^® (cabozantinib) for adult patients with unresectable or metastatic, well differentiated pancreatic (pNET) and extra-pancreatic (epNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues.

NEW YORK, July 16, 2025 (GLOBE NEWSWIRE) -- Aptorum Group Limited (NASDAQ: APM) ("Aptorum Group," “Aptorum” or the "Company"), a clinical stage biopharmaceutical company dedicated to addressing unmet medical needs in oncology  and infectious diseases, and DiamiR Biosciences (“DiamiR”), a developer of proprietary innovative blood-based tests for brain health and other diseases with a CLIA licensed, CAP accredited clinical laboratory in New Haven, CT, today announced that they have entered into a definitive agreement for an all-stock merger transaction, in which DiamiR Biosciences will retain its name and become a wholly-owned subsidiary of Aptorum Group upon consummation of the merger.The combined company expects to remain listed on the Nasdaq Stock Market following the closing of the merger.