Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy

Next Previous
X
Search
Trial Profile

A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 06 Nov 2021

Price : $35 *
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs Ataluren (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions
  • Sponsors PTC Therapeutics

    • Most Recent Events

    • 12 Aug 2020 The number of primary end-points is increased from 3 to 4 (3 newly added). The duration of treatment and follow up phases has been changed fram 4 weeks and 48 weeks to 52 weeks and 4 weeks respectively, also changing the duration of total study from 52 to 60 weeks. Treatments has been modified to included weight assessment of all participants in every 12 weeks.
    • 09 Jul 2018 Results at week 28 published in the PTC Therapeutics Media Release
    • 09 Jul 2018 According to a PTC Therapeutics media release, the data was presented at the International Congress on Neuromuscular Diseases 2018.

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top