Medical

Pathways Neuro Pharma Targets Rare Pediatric Neurological Disorders with Pioneering Gene Therapy

WEST CHESTER, PA — Pathways Neuro Pharma Inc. has announced a significant pivot in its development pipeline, prioritizing rare pediatric neurological disorders, with Juvenile Parkinson’s Disease (JPD) leading the charge. This …

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Ocugen

Ocugen’s OCU400 Receives ATMP Classification from European Commission, Advancing Retinitis Pigmentosa Treatment

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has reached a significant milestone in the development of its gene-agnostic therapy, OCU400, as the European Commission has delivered a positive opinion designating it …

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gene

Revolutionary Gene Therapy Approved: Kebilidi Offers New Hope for Rare Genetic Disorder

WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq), marking the first gene therapy sanctioned for the treatment of aromatic L-amino …

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