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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Allison Moore is going to be published!

HNF’s CSO (Chief Scientific Officer), Sean Ekins wrote a blog about his work with Allison Moore and her two “fighter Mom” friends Lori Sames and Jill Wood. He named his blog: “Rare disease heroes – Extraordinary collaborators we should be listening too.” Sean helped my friends and I write a paper called: “Multifaceted roles of ultra rare and rare disease patients/parents in drug discovery.” YES, Allison Moore is going to be published! It will be in Drug Discovery Today, soon. The link to the reprint is below.

HNF-WVU-NIOSH Study update:

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Dr. Robert Baloh Answers Our Questions About iPS Cell Research

Big news is happening on a cellular level at Cedars-Sinai Medical Center’s Regenerative Medicine Institute in Los Angeles, CA.  Robert H. Baloh, MD PhD, Director of Neuromuscular Medicine, in conjunction with Patrick D. Lyden, MD Neurology Dept. Chair, and Clive Svendsen, PhD, Institute Director, are studying disorders that start in nerve cells. 

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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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