Global Genes 2017 RARE Patient Advocacy Summit
Marie Wood and Erin Ward represented MTM-CNM Family Connection, Inc. at the Global Genes 2017 RARE Patient Advocacy Summit in Irvine, CA this September. They spent the two days of the Summit attending a wide range of patient advocacy sessions, including understanding the drug approval process, accelerating clinical pathways, partnering with industry, networking with other rare disease communities, and many more. It was an amazing feeling knowing many of the therapeutic strategies discussed throughout the event are also avenues being pursued in our MTM-CNM Community, including gene therapy (Audentes), antisense oligonucleotides (Dynacure), repurposing of existing drugs (Dr. Dowling), and enzyme replacement therapy (Valerion).
A wonderful moment at the Summit was when Barbara Wuebbels, VP of Global Patient Relations at Audentes received the 2017 RARE Champion of Hope Legacy in Advocacy Award! Thank you Barbara for your work with our MTM community over the last few years and for your extraordinary contributions to the global rare disease community throughout your career.
We were happy to help represent the MTM-CNM community voice at the Summit and look forward to incorporating all that we learned into MTM-CNM Family Connection's continued work for our community. If you'd like to read more about the wonderful work of Global Genes and the Rare Patient Advocacy Summit,or view some of the recorded sessions, please click the button below: