Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S.

FDA grants Breakthrough Therapy designation to luvesilocin for postpartum depression following positive results from the phase 2 RECONNECT trial.

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Neurogene received FDA Breakthrough Therapy Designation for NGN-401 in Rett Syndrome, driving a >25% share price surge. Click ...

Larimar on Tuesday said nomlabofusp, a protein replacement therapy that is being developed as a potential treatment for Friedreich's ataxia, was granted the important designation that is designed to ...

Breakthrough Therapy Designation Based on Positive RECONNECT Phase 2 Clinical Data ---- Aligned with U.S. Food and Drug Administration on ...

Here’s something big: scientists in China have actually reversed type 2 diabetes using stem-cell therapy. It’s a major step ...

Ongoing Phase 3 full approval clinical trial of ateganosine holds high probability of technical success for interim and full analysisFDA Fast ...

A groundbreaking topical gene therapy for 'butterfly skin disease' has transformed rare disease treatment. Suma Krishnan's ...

Forty-Hz auditory stimulation increased amyloid clearance markers in aged primates and produced lasting effects, supporting its potential as a non-invasive Alzheimer’s therapy. Scientists at the Kunmi ...

Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.