Israeli biotech firm Protalix Biotherapeutics Ltd. (PLX: News ) is only months away from announcing the late-stage study results of its lead product candidate prGCD for the treatment of Gaucher (pronounced Go-shay) disease. Gaucher disease is an inherited genetic disorder, in which patients lack the normal form of the glucocerebrosidase,...
treatment News
CARMIEL, Israel and BOSTON – Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthare Group (Chiesi Group),...
CARMIEL, Israel and BOSTON – Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group),...
RICHLAND, Wash. — Doctors have nearly a dozen new targeted drugs to treat patients with acute myeloid leukemia, or AML, yet three of four patients still die within five years. Some patients succumb within just a month or two, despite the battery of drugs used to treat the aggressive blood...
DUBLIN, Ireland — Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today announced the publication related to the mechanism of action, pharmacological characteristics, and clinical utility of birtamimab, a potential best-in-class anti-amyloid treatment for AL amyloidosis. The...
TAMPA, Fla. — Psilera, a leading biopharmaceutical company focused on developing novel treatments for neurological disorders, is pleased to announce the selection of behavioral variant frontotemporal dementia (bvFTD) as the lead indication for its groundbreaking drug candidate, PSIL-006. This devastating form of early-onset dementia currently lacks approved treatments to manage symptoms...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that both the United States Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for PTC923 for the treatment of patients with hyperphenylalaninemia. Phenylketonuria (PKU) accounts for 98% of all hyperphenylalaninemia cases and is a metabolic condition that...
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. “The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe,” said Matthew B....
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. FCS is a rare genetic disease which results in significant disease burden to patients including potentially fatal pancreatitis...
SOUTH PLAINFIELD, N.J. — PTC Therapeutics, Inc. (PTC) today announced the receipt of two government grants to support clinical development programs in rare disorders. The U.S. Department of Defense Neurofibromatosis Research Program has awarded an $822,345 grant to support a new open-label Phase 2 clinical trial of PTC’s product candidate...