These are Maple Syrup Urine Disease, Methylmalonic Acidemia/Propionic Acidemia, Galactosemia, Phenylketonuria, Gaucher ...

Human genetics biotech Maze Therapeutics has navigated the biotech world’s tricky fundraising landscape to land a $115 ...

Shares of PTC Therapeutics PTCT rose nearly 19% on Monday after announcing that it has entered into an exclusive global ...

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data. PKU is a rare ...

RBC Capital upgraded PTC Therapeutics (PTCT) to Outperform from Sector Perform with a price target of $63, up from $39. The firm views today’s ...

Learn more about whether BioMarin Pharmaceutical Inc. or Exact Sciences Corporation is a better investment based on AAII's A+ ...

Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect ...

At the center of a new licensing deal is an experimental medicine, PTC518, which is currently being tested in a roughly ...

Novartis will pay PTC Therapeutics up to $2.9 billion in a licensing deal for its Huntington's treatment. PTC Therapeutics stock surged.

A groundbreaking drug, a massive deal, and a stock rally--this could reshape neurodegenerative disease treatments.

Symposia Title Description Speaker Time, Location Communicating a Diagnosis of Dravet Syndrome (DS) to Parents/Caregivers: An International ...