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...
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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...
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 it submitted a New Drug Application (NDA) for RECORLEV® (levoketoconazole) for the treatment of endogenous Cushing’s syndrome to...
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 U.S. Food and Drug Administration (FDA) has accepted for review the Company’s New Drug Application...
New York, NY – Structural insights into a potent antimalarial drug candidate’s interaction with the malaria parasite Plasmodium falciparum have paved the way for drug-resistant malaria therapies, according to a new study by researchers at Weill Cornell Medicine and Van Andel Institute. The antimalarial molecule, TDI-8304, is one of a new class...
PHILADELPHIA – How molecules of the oldest branch of the human immune system have interconnected has remained a mystery. Now, two new structures, both involving a central component of an enzyme important to the complement system of the immune response, reveal how this system fights invading microbes while avoiding problems...
Munich, Germany – Many important medicines, such as antibiotics and anticancer drugs, are derived from natural products from Bacteria. The enzyme complexes that produce these active ingredients have a modular design that makes them ideal tools for synthetic biology. By exploring protein evolution, a team led by Helge Bode from...