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...
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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,...
Football, faith and family play very important roles in the life of Davidson College linebacker Storm Monroe. Storm was just 9 years old in 2010 when he was diagnosed with Guillain-Barre syndrome. GBS is a rare disorder in which the body’s immune system attacks the nerves, usually resulting in weakness...
New Brunswick, NJ – Rutgers researchers can predict which patients will benefit from a popular prostate cancer drug – and have devised a strategy that may make the treatment work longer. “This work should help doctors know which patients’ prostate cancers will and won’t respond to the androgen deprivation therapy...
Immune checkpoint inhibitors can boost the immune system’s response to tumor cells, but the medications tend to be ineffective against certain cancers, especially colorectal and pancreatic cancers. A new clinical trial led by investigators at Massachusetts General Hospital (MGH) indicates that combining radiation with immune checkpoint inhibitors may be an...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented real-world results from the STRIDE patient registry demonstrating that treatment with Translarna™ (ataluren) delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care (SoC) alone. Pulmonary function decline was...
Researchers studying the bleeding disease hemophilia in mice have increased the rodents’ ability to produce a crucial blood-clotting protein, a new study says. Their success may pave the way for human trials. Past studies have successfully introduced a gene into animals’ cells to make the clotting protein, Factor VIII, which...
Dunedin, New Zealand – People suffering from Long COVID or Myalgic Encephalomyelitis /Chronic Fatigue Syndrome (ME/CFS) could benefit from a coordinated treatment strategy, a new University of Otago study has found. The pilot study, published in Nature Portfolio’s Scientific Reports, an open-access journal, has confirmed what researchers have suspected for...
DUBLIN, Ireland and TREVOSE, Pa. – Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,020,393 entitled, “Methods...
DUBLIN, Ireland and TREVOSE, Pa. – Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced that post hoc analyses from a one year open-label study evaluating daily use of KEVEYIS® (dichlorphenamide) for the treatment...