Do you have questions for companies working on MTM/CNM?

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At the 2019 MTM-CNM Family Conference, we will have representatives from two biotechnology companies that are working to develop therapeutic interventions for our MTM-CNM community.

1) Audentes Therapeutics is working on AAV-based gene therapy for X-linked Myotubular Myopathy: https://www.audentestx.com/x-linked-myotubular-myopathy/

2) Dynacure is working on ASO-based therapy for X-linked (MTM1), autosomal dominant (DNM2), and autosomal recessive (BIN1) forms of Centronuclear Myopathy: https://www.dynacure.com/pipeline/dyn-101/

Both companies would like to hear your questions and respond as best as they can at our Conference. Please help us compile some questions that will be shared with the companies ahead of the Conference. This will help them prepare their speakers and content to best meet the needs and interests of our community.

Your personal information will not be recorded. The questions will be summarized and presented without any identifying information.

While we hope you will be joining us in person at the Conference, you are welcome to submit questions even if you are not able to be there.

My questions for Audentes:

My questions for Dynacure:

Any other questions I have:

I am planning to attend the 2019 MTM-CNM Family Conference *

(this response will be used to help clarify any differences in questions between those in attendance and those not present, to ensure all voices are included)

Yes

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