Tag: mortality

Allschwil, Switzerland, July 29, 2025

July 28, 2025
News Release

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®.

Hennigsdorf/ Berlin, July 23, 2025 – 4TEEN4 Pharmaceuticals GmbH today announced that the first patient has been dosed in a Phase 1b/2a PROCARD1 clinical trial evaluating its monoclonal antibody, procizumab, in patients with shock caused primarily by cardiogenic events. Shock is a life-threatening condition characterized by a sudden breakdown in circulatory function due to end-stage disease or acute events, which often leads to organ failure. With only symptomatic treatment options available, shock is associated with a high mortality rate of over 50%. The Phase 1b/2a trial is designed to identify a target dose for procizumab in preparation for further clinical development. In addition, the trial will provide signals of efficacy. In a Phase 1 study in healthy volunteers, procizumab was well tolerated.

ORION CORPORATION
PRESS RELEASE
21 JULY 2025 at 10:30 EEST          

ORION OYJ
LEHDISTÖTIEDOTE
21.7.2025 klo 10.30

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –

– EZMEKLY is the first and only therapy to receive marketing authorization in the EU for both adults and children (≥2 years) with NF1-PN, a rare genetic disorder with debilitating symptoms –

Multiple Tobacco Brands Partnering with 22nd Century to Expand Availability and Awareness of VLN Based Reduced Nicotine Content Products