PTC Therapeutics Earns FDA Nod for SEPHIENCE™: A New Hope for Phenylketonuria (PKU) Patients

PTC Therapeutics has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of SEPHIENCE™ (sepiapterin), marking the first and only therapy approved for broad treatment of…

Continue Reading PTC Therapeutics Earns FDA Nod for SEPHIENCE™: A New Hope for Phenylketonuria (PKU) Patients

FDA Advisory Panel Delivers Mixed Verdict on Blood Disorder Drugs and PureTech CEO Steps Down

In a week of significant developments for the biopharma industry, an FDA advisory committee issued pivotal recommendations on two high-profile blood disorder drugs, while PureTech Health announced a leadership change…

Continue Reading FDA Advisory Panel Delivers Mixed Verdict on Blood Disorder Drugs and PureTech CEO Steps Down

Acknowledgement: Patient Worthy is honored to be a media partner of the National Organization for Rare Disorders (NORD).  The mission of NORD is to improve the health and well-being of…

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