Category: PTC Therapeutics

  • Listening to ultra-rare disease communities

    When it comes to ultra-rare diseases, the smaller the patient population, the bigger the challenges. As a global commercial biopharmaceutical company pioneering therapies for ultra-rare diseases, PTC Therapeutics is familiar with the challenges. The company prides itself on a patient-centric approach to treatment but there can be significant hurdles in patients accessing potentially life-changing treatments. […]

  • Scotland opens door to Translarna for Duchenne muscular dystrophy

    A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics’ Translarna for the first time.  The Scottish Medicines Consortium (SMC) says that Translarna (ataluren) can be provided by NHS Scotland for a period of three years, once PTC […]

  • PharmaShots Weekly Snapshot (Aug 17 -21 2020)

    1. BioMarin Reports NDA Submission to the US FDA for Vosoritide to Treat Children with Achondroplasia Published: Aug 20, 2020 | Tags:  BioMarin, Reports, NDA, Submission, US, FDA, Vosoritide, Children, Achondroplasia 2. Junshi and Impact to Establish Joint Venture for Senaparib (IMP4297) in China Published: Aug 20, 2020 | Tags:  Junshi, Impact, Establish, Joint Venture, […]