Submit your personal stories about Ataluren/Translarna and help our friends in the USA

Duchenne UK and Action Duchenne are working together to collate the testimonies of Duchenne families in the UK who have had experience of being involved in the clinical trial of Ataluren/Translarna, or who have received the drug under the Managed Access Programme.

This is an important announcement to ask all UK families who participated in the Ataluren/Translarna trials and/or are currently receiving Ataluren/Translarna if they would like the opportunity to submit their personal experiences to the Food and Drug Adminsitration (FDA) in the United States. The FDA is a regulator that decides on whether new medicines should be approved for use in the USA.

Later this month, the FDA will host what’s called an Advisory Committee Meeting for PTC’s therapy Ataluren, on Thursday 28 September 2017. This meeting is also known as an Adcomm.

Our friends at PPMD in the United States have been working hard with the Duchenne community to prepare for the upcoming meeting, and they need our help to gather personal testimonies from the families in the UK.

Duchenne UK, with our partner patient organisations in the UK, fought a long campaign to have Translarna made available to Duchenne patients in the UK through a Managed Access Agreement. We could not have done this without your support.

We would love for our Duchenne families in the UK who have experiences with Ataluren/Translarna to share their personal story with the FDA Advisory Committee.

Please consider submitting a written testimony. We are working with Duchenne UK to make sure our voices are heard by the FDA. We have already collated a lot of testimony, but the more of your stories we have, the more powerful our message will be.

Please send your written testimonies by email to: [email protected]

  • The FDA has requested that all written submissions may be submitted to the docket either electronically or by mail on or before 27 September 2017. Comments submitted to the docket by 14 September will be shared with the Advisory Committee, after that comments will be considered by the Agency. Please consider sending us your story by 11 September so we can work on putting them together for a submission.

Here are some things to think about when drafting your story. It should not be longer than one page.


Points for consideration:

For those with experience on the product:

  • What physical changes have you seen as a result of the experimental product (in this case Ataluren/Translarna)?
  • What activities of daily living did you/your child have difficulty performing prior to the trial’s initiation that became easier or possible as the trial went on?
  • Are there people in you/your child’s daily life who may have been unaware of the trial and made observations that spoke to increased endurance and stamina or function? (i.e. teachers, family members, etc)
  • If at any point during the trial you/ your child experienced side effects related to the experimental product, describe those – and discuss whether you considered them to be severe and how that played into your decision to continue/discontinue participation in the trial. If you have not seen any side effects please indicate that. 
  • Describe how Duchenne impacts you/your child and your family

What is the FDA Advisory Committee?

  • The Peripheral and Central Nervous System Drugs Advisory Committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs.
  • Part of the FDA mission is to evaluate new therapies and determine which are safe and effective for their intended use.
  • The Committee is advisory in nature, providing recommendations to the agency; FDA makes the final decisions.
  • The Peripheral and Central Nervous System Drugs Advisory Committee is one of 48 technical and scientific advisory committees and panels; it would be responsible for reviewing potential treatments for Duchenne.

In the UK we have The European Medicines Agency (EMA) which has the same responsibilities as the FDA in America, it is a decentralised agency of the European Union (EU), located in London. The Agency is responsible for all the scientific evaluation, supervision and safety monitoring of medicines in the EU.

For more information please visit PPMD website….



If you have any questions please do not hesitate to ask either DUK, AD or PPMD. 

Email – [email protected]gary@actionduchenne.org or Ryan@parentprojectmd.org

Published on 9 April 2017

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