SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of a manuscript, “Gene Therapy in the Putamen for Curing AADC Deficiency and Parkinson’s Disease,” in the European Molecular Biology Organization Journal. The paper describes a pioneering approach that delivers gene therapy to a specific part of the brain...
treatment News
OSLO, Norway – Targovax ASA (OSE: TRVX), a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, today announce that the European Patent Office has granted EU Patent no EP3402889. The patent covers the use of ONCOS-102 in combination with chemotherapy in malignant pleural mesothelioma. Torbjørn Furuseth,...
Basel, June 5, 2009 – New data show that at 12 months newly diagnosed patients taking Tasigna® (nilotinib) to treat a life-threatening form of chronic myeloid leukemia (CML) had rapid responses and a deep reduction in the amount of the abnormal protein that causes this disease. “Tasigna now demonstrates potential...
CTA approved by Health Canada in November 2021; IND approved and open in 2019 Queen’s University selected as initial clinical site under the direction of Dr. Jagdeep Wahlia, principal investigator PPT1 enzyme activity of 5% or greater normalizes survival and significantly improves clinical phenotype based on natural history data Preliminary...
DALLAS, TX — Taysha Gene Therapies, Inc., a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that it will present clinical data on its TSHA-102 program in evaluation for Rett syndrome during...
DALLAS – Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced publication of new preclinical data for TSHA-104 in...
DALLAS, TX — Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today announced the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced...
DALLAS – Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has been granted orphan drug...
AyuVis Research Inc., a TechFW client, has been granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its small molecule drug AVR-48 for the prevention of bronchopulmonary dysplasia or BPD. AVR-48 works to modulate the immune system by decreasing the inflammatory response to MV and oxygen...
A unique and innovative telemedicine project is providing distant nursing home patients with Parkinson’s disease access to neurologists at the University of Rochester Medical Center. A pilot study of the project – the results of which were released this month at the International Congress of Parkinson’s Disease and Movement Disorders...