BUFFALO, NY- February 7, 2024 – A new research paper was published in Aging Volume 16, Issue 2, entitled, “PROX1 interaction with α-SMA-rich cancer-associated fibroblasts facilitates colorectal cancer progression and correlates with poor clinical outcomes and therapeutic resistance.” The tumor microenvironment (TME) plays a vital role in tumor progression through...
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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...
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1...
TORONTO, Canada – PTC Therapeutics Canada ULC announced that Health Canada has approved Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month old and older with sepiapterin-responsive PKU “We’re...
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. (NASDAQ: PTCT) today introduced a new no-charge, patient-initiated testing program in the United States for individuals who have symptoms consistent with, or a diagnosis of, cerebral palsy (CP) with no evidence of brain injury. CP is a group of disorders that affect a person’s ability to...
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the...
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...