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40 US Physicians Submit Letter to FDA Encouraging Approval of Ataluren; Cite DuchenneConnect Registry Data

PPMD thanks the 40 US physicians joining the call for approval of ataluren. This letter compliments the international expert letter recently sent to the FDA. The US letter cites data from PPMD's DuchenneConnect Registry, the largest global Duchenne patient registry, to compare loss of ambulation and make the case for approval. Thank you to everyone who has contributed data and joined the registry. Patient reported data matters and can aid in the effort to better characterize Duchenne.

Read the US letter

Read the international letter

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April 2018
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