October 23, 2020

Solid & Ultragenyx Announce Strategic Collaboration to Develop & Commercialize New Gene Therapies for Duchenne

We are pleased that Solid Biosciences has announced a strategic collaboration and license agreement with Ultragenyx Pharmaceutical. Emil Kaakis, CEO of Ultragenyx, has been focused on rare diseases across his career and has considerable experience…

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October 1, 2020

FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

PPMD is delighted to learn that the FDA has lifted the hold on Solid Biosciences’ IGNITE-DMD clinical trial for their gene therapy product (SGT-001) delivering a micro-dystrophin transgene via AAV9. Solid has worked diligently to…

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July 24, 2020

Solid Biosciences Provides Update Regarding the IGNITE DMD Phase I/II Clinical Hold

Tonight at PPMD’s Virtual Annual Conference, Solid Biosciences shared that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. They also issued a press release here. During this hold, Solid will continue to…

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May 7, 2020

Solid Biosciences Shares Update on SGT-001 Phase I/II Clinical Hold for IGNITE DMD Program

Solid Biosciences shared today that their IGNITE DMD Phase I/II clinical trial will remain on clinical hold. Solid will continue to work with the FDA to provide further data and analyses relating to their manufacturing…

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March 30, 2020

Solid Biosciences Shares Letter to the Duchenne Community: COVID-19 Update

Solid Biosciences Inc. has shared an update with the community regarding the IGNITE DMD clinical hold and COVID-19. Solid’s Letter to the Duchenne Community: Letter to the Duchenne Community: COVID-19 Update Dear Duchenne Community, During…

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March 12, 2020

Solid Biosciences Provides Letter to the Duchenne Community: Data and IGNITE DMD Clinical Trial Update

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that biomarker data from all three patients dosed in the 2E14 vg/kg cohort of IGNITE DMD…

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December 18, 2019

Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that the status of the sixth patient dosed in the higher 2E/14 vg/kg, who experienced the…

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November 12, 2019

Solid Biosciences Provides SGT-001 Program Update

Solid Biosciences Inc. provided a clinical update on SGT-001 and reported that the FDA has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. To date,…

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November 4, 2019

End Duchenne Tour Recap: Memphis, TN

PPMD’s End Duchenne Tour made a stop in Memphis, TN on Saturday, October 26. We were joined by over 60 people,  including families, an amazing group of volunteers in our kids room, six members of…

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September 24, 2019

End Duchenne Tour Recap: Wilmington, Delaware

PPMD’s End Duchenne Tour made a stop in Wilmington, Delaware on September 14. We were joined by over 100 people, including 30 families, 20 healthcare professionals and researchers, 15 industry partners, and 5 members of…

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