NEEDHAM, Mass.— Stealth Biotherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for elamipretide for the treatment of Barth syndrome....
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BUFFALO, N.Y. – University at Buffalo research has identified how a misstep in the genesis of a key component of the kidney causes infantile cystinosis, a rare disease that significantly shortens the lifespan of patients. Published in the International Journal of Molecular Sciences, the work reveals that the mechanisms that...
Madison, Wisconsin – Technology developed at the University of Wisconsin–Madison to grow “rosettes” of brain and spinal tissue gives scientists new ways to study the growing human brain, including a recent study of how genetic mutations linked to autism affect early stages of human brain development. It’s the latest discovery...
Hoboken, NJ – Cell therapy represents a potential regenerative treatment for osteoarthritis. A recent analysis of all relevant published studies indicates that stem cell transplantation from different sources is effective for treating knee osteoarthritis, the most prevalent chronic joint disease. The review and meta-analysis, which is published in the Journal...
Vancouver, Canada – An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH). “This is a significant step toward a...
Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage. Duchenne primarily affects males and is usually diagnosed by age 4. While a variety of therapies can slow progression and extend life expectancy, the disease has...
STETSON, Maine — On a back road in the rural town of Stetson, a bright fuchsia and green RV is hard to miss, standing out against Maine’s bleak November scenery. Printed on the side of this van are a few inspirational quotes, and in larger letters are the words “Whitney...
BEDFORD, Mass. – Stoke Therapeutics, Inc, a new company working to increase gene expression to treat a wide array of diseases caused by genetic insufficiency, announced that it has completed a $40 million Series A financing to support the further advancement of multiple pre-clinical development programs. The Series A round...
BEDFORD, Mass. – Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate...
BEDFORD, Mass. – Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced highlights from presentations of clinical data at the American Epilepsy Society (AES) 2023 Annual Meeting December 1 – 5, in Orlando,...