CATEGORIES: Research
rare cast

How One Rare Disease Organization Used Technology to Provide Greater Patient Insight to FDA

by | Nov 19, 2018 | 1 comment

Daniel Levine from Global Genes spoke with HNF’s CEO and Founder Allison Moore on this edition of Global Genes Rare Cast about how HNF used a technology company to gather patient information to present at the Patient-Focused Drug Development Meeting with the FDA.

CMT can impact the ability for patients to type or write. By joining forces with a technology company, HNF was able to gather patient feedback using voice technology, allowing patients to give their comments over the phone through a simple automated system.

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Hot Off the Press – Potential Treatment for CMT1A

Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.

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1 Comment

  1. David Beckingham

    Love what you’re doing and your progress. I don’t know how, other than politically, to help get Pharnext approved in Canada. Obviously it is easier once a drug has FDA Approval. I have a son who could be in clinical trials. I had DNA Testing which confirmed I have CMT1A so we assume he does also. Will the drug be affordable in the US and Canada?

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