Health Canada approved AGAMREE for the treatment of Duchenne muscular dystrophy in patients aged 4 years and older This approval makes AGAMREE the first and only approved therapy for DMD in Canada October 3, 2025 – Santhera Pharmaceuticals (SIX: SANN) today notes that Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older following Priority Review, marking the first approved treatment for the disease in Canada.

Oct. 02, 2025 -- Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary, investigational therapy for the treatment of patients with retinitis pigmentosa (RP). “Breakthrough Therapy Designatio

Additional pre-planned analysis of the NCX 470 Denali trial completed NCX 470 maintains robust intraocular pressure lowering at 6, 9 and 12 months Other analyses broadly in line with the trends seen for Mont Blanc Data presentations planned for upcoming ophthalmology conferences October 2, 2025 – Nicox SA (Euronext Growth Paris: FR0013018124, ALCOX), an international ophthalmology company, today announced that it has completed the additional pre-planned analyses of the NCX 470 Denali Phase 3 cl

Biolab Farmacêutica gains exclusive rights in Brazil to GMRx2, an innovative single pill combination of three medicines, including two doses that are lower than those currently available in single pill combinations, designed to deliver the blood pressure-lowering benefits of triple combination therapy early in the treatment pathway, with an established safety profile George Medicines receives upfront licensing fee and is eligible for regulatory and commercial milestone payments, as well as a fut

Cirrus’ lead program is a novel AAV gene therapy designed to restore IRAK-M, a central regulator of the eye’s immune homeostasis, so as to prevent AMD progression and preserve central vision IRAK-M levels decrease with age and more prominently in people with dry AMD, presenting an important therapeutic target to combat this leading cause of vision loss in people over 50 Seed funding will advance the lead program into IND-enabling studies and support a growing pipeline of next-generation ocular m

Oct. 01, 2025 -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) and its majority-owned subsidiary, Cyprium Therapeutics, Inc. (“Cyprium”), today announced that the U.S. Food and Drug Administration (“FDA”) has issued a Complete Response Letter (“CRL”) relating to the New Drug Application (“NDA”) for CUTX-101 (copper histidinate), intended to treat Menkes disease in pediatric patients. In December 2023, Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-ow

AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue recruiting in our STELLA observational study Oct. 02, 2025 --- AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted AAVB-039, the company’s investigation

ANAVEX3-71-002 trial achieved its primary endpoint demonstrating safety and tolerability in both male and female adults Oral ANAVEX®3-71 tablet exhibits superior pharmacokinetics compared to the current immediate-release oral capsule, enabling once-daily dosing NEW YORK, Oct. 02, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkins

Oct. 01, 2025 -- RetinalGenix Technologies Inc. OTCQB:RTGN (“RetinalGenix” or the “Company”), a pioneering developmental-stage company focused on ophthalmic screening, monitoring, pharmacogenetic mapping, and repurposed drug development for early detection and treatment of eye and systemic diseases, and RGEN Inc., a leader in genetic research and innovation (“RGEN”), today announced a strategic partnership to deliver game-changing advancements in the early detection and risk prediction of Advan

Oct. 01, 2025 -- Aerska, a biotechnology company aiming to redefine the treatment of neurological diseases, today announced it has raised $21 million in seed financing, co-led by Age1, Backed VC and Speedinvest, with participation from Blueyard, Lingotto (Exor), Norrsken VC, Kerna, PsyMed, Saras, and Ada Ventures. The company is developing systemically administered RNA interference (RNAi) medicines designed to silence genes that drive brain diseases. Delivering genetic medicines to the brain