Tag: neurons

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®.

New products reflect Mikra’s commitment to recently unveiled Mind-First Movement prioritizing mindset as the primary driver of peak performance

NEWTON, Mass., July 10, 2025 (GLOBE NEWSWIRE) -- Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS), a clinical-stage biopharmaceutical company developing a novel therapeutic that targets soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD), will present new findings at the upcoming Alzheimer's Association International Conference (AAIC®) 2025 in Toronto. The presentations include a cost savings analysis of the use of pTau217 as a screening tool in Acumen’s Phase 2 ALTITUDE-AD trial of sabirnetug as well as a nonclinical study evaluating the relative selectivity of sabirnetug to targeting AβOs versus Aβ monomers. The conference will be held July 27-31, 2025, both in-person and online.

Rincell-1 addresses a multi $Bn global market opportunity  where no disease modifying treatments currently exist

CAMBRIDGE, Mass., June 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). These latest findings from Part C of the DEVOTE trial evaluating a higher dose regimen of nusinersen and the NURTURE trial which evaluated the approved 12 mg regimen (SPINRAZA^®) in clinically presymptomatic SMA were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. Biogen’s applications for the higher dose regimen of nusinersen are currently under review in the U.S., Europe, Japan and other global markets. The higher dose regimen of nusinersen comprises a more rapid loading regimen – two 50 mg doses 14 days apart – and a higher maintenance regimen – 28 mg every four months.

Cognitive Wellness Announcement Highlights New Senior Brain Health Formulation with Scientifically Backed Ingredients

SAN FRANCISCO, June 20, 2025 (GLOBE NEWSWIRE) -- Structure Therapeutics Inc. (NASDAQ: GPCR), a clinical-stage global biopharmaceutical company developing novel oral small molecule therapeutics for metabolic diseases, today announced two upcoming late-breaking poster presentations at the American Diabetes Association 85^th Scientific Sessions, being held from June 20-23 in Chicago, IL.