With positive Phase 1 results for the metabolic disorder drug MZE782, Maze Therapeutics plans to advance the small molecule to Phase 2 tests in phenylketonuria and chronic kidney disease. Following the data readout, Maze raised $150 million in a private placement.
Otsuka Pharmaceutical is acquiring Jnana Therapeutics, whose lead drug is a potential treatment for the rare metabolic disorder phenylketonuria (PKU). Jnana’s drug could provide an alternative to two FDA-approved PKU medicines from BioMarin Pharmaceutical.
Choosing the right brain injury rehabilitation program is a critical decision that can shape the future for you or your loved one.
PTC Therapeutics plans to talk to regulators about regulatory submissions for sepiapterin after the drug met the main goal of a Phase 3 study. The small molecule is a potential treatment for phenylketonuria, a rare enzyme deficiency.
A collaboration between Nvidia and startup Evozyne was able to produce novel versions of a human protein never before seen in nature but with enhanced function and the same safety as native protein. The research lays the groundwork for potential new therapies for a rare inherited disorder.
A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed cancer.
The company plans to enter the clinic by the close of 2016 with a red blood cell-based therapy that treats phenylketonuria.
Incorporating aquatic therapy can enhance patient outcomes and shorten recovery times for health care professionals. In the Chicago suburbs, the demand for such programs is rising.