October 14, 2016
Cambridge, MA

Patients At The Center: A Note From Two Mothers

It has been an exciting and emotional time for our Duchenne muscular dystrophy (DMD) community. Progress in science and on the regulatory front fills us with hope for the future and further reinforces our mission to discover and develop effective and safe treatments for all patients with DMD.

As mothers of boys with DMD, we acutely understand and live with this monster that is DMD. Solid Biosciences is working to address the entirety of the problem, recruiting top talent, forming strong partnerships, advancing only the best therapeutic candidates, engaging with regulators early, and developing and validating better biomarkers and clinical endpoints. 

However, this work cannot be done without insight from those who are closest to the disease – patients and their families. That is why we chose to be the champions of Patient Advocacy at Solid.

It is our responsibility and passion to ensure that the patient voice and perspective is part of the company’s DNA, woven into every step of our research, development and clinical programs. To get our work done right, we must engage early and often with the DMD community.

We are so proud of Solid’s progress over the past quarter, much of which could not have been done without the support of the community. We are growing in both science and talent to support our 360-degree approach to solving DMD. We are on track to begin clinical studies with our lead gene therapy candidate in 2017, have grown our pipeline with additional preclinical candidates and built out an entire platform dedicated to testing and validating new and improved biomarkers that, we hope, will benefit the entire field working on DMD therapies. We also opened an office in London, U.K. to build our clinical team and support our commitment to Europe. And, finally, we are excited to continue to build DMD charity partnerships, including those with Alex’s Wish and Duchenne Now.

(Left) Annie Ganot with 6 year old Eytani. (Right) Kerry Rosenfeld with 14 year old Gavriel

We are deeply committed to our DMD community, which is the very heart of what we do. We are looking forward to seeing many of you at the upcoming Action Duchenne Conference in London!

Annie Ganot, Head of Patient Advocacy

Kerry Rosenfeld, Patient Advocacy, Europe                                            

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The Duchenne Research Fund (DRF) provided Solid with a research grant of $600,000 to support its LTBP4 program to combat fibrosis and to support novel biomarker work in Duchenne muscular dystrophy (DMD).


Solid Biosciences congratulates Sarepta on the U.S. approval of the first therapy for Duchenne muscular dystrophy.


Solid was proud to sponsor the 8th Annual Gals For Cal team at the recent Max Performance Title 9 Triathlon at Hopkinton State Park in Massachusetts.


Solid Biosciences joined the Jett Foundation and other members of the local Cambridge, MA community to observe World Duchenne Awareness Day.


Solid Biosciences Community Update Q2 2016


Grants from the charities Duchenne Now, Save Our Sons and Fight DMD will support important work in furthering Solid’s gene therapy and disease modifying therapy programs.


The biologic candidate LTBP4 has the potential to promote muscle health and reduce fibrosis in patients with Duchenne muscular dystrophy.


Carl Morris, Solid Biosciences’ vice president of Research and Development, speaks about being a “Game Changer in Rare Disease.”


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