WASHINGTON, DC / ACCESSWIRE / October 9, 2022 / America's currency would regain stable footing for the first time in half a century if a bill just introduced by U.S. Representative Alex Mooney (R-WV) becomes law.
CAMBRIDGE, Mass., Sept. 20, 2021 /PRNewswire/ -- Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced positive data from clinical studies evaluating both SYNB1618 and SYNB1934, investigational Synthetic Biotic™ medicines for the treatment of phenylketonuria (PKU).
After years chasing a hard-to-drug group of targets called solute carriers, Jnana Therapeutics is revealing its lead program and a new $50 million financing to get it into the clinic.
BioMarin Pharmaceutical announced that it has dosed the first participant in the global PHEARLESS phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with Phenylketonuria (PKU). BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.
European Medicines Agency (EMA) publishes information on excipients for labeling & package leaflet of human medicines
A judge has ordered NHS England to reconsider after refusing to fund treatment with Biomarin's Kuvan for a boy with the rare genetic disorder phenylketonuria (PKU).
One of the oldest PKU patients to participate in the trial, Lester has encouraged others with his condition to return to KCH to receive the progressive treatment, which he credits with allowing him to have more flexibility in his diet. Dr. Stephen Amato, a genetic metabolic specialist at KCH who serves as the principal investigator on the clinical trial, and his study team faced the logistical challenge of bringing adult patients back to KCH to participate in the trial. After adjusting to their condition as children, many PKU patients treated at KCH as children drifted away from their original point of care. Fewer than 20 percent of PKU patients nationwide receive regular clinical care, and even fewer follow the restrictive diet as adults.
Recall: Baxter`s Clinimix (2015-06-19)