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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We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD.


- Activities to Resume Enrollment in IGNITE DMD Phase I/II Clinical Trial are Underway

- Conference Call Scheduled for Today at 8:30 A.M. ET


- Data Reinforce Potential of SGT-001 as an Important Treatment Candidate for DMD -

- Company Continues to Advance Gene Therapy Portfolio -

Company Finalizing Response to FDA Regarding Clinical Hold on SGT-001 Phase I/II Clinical Trial

Letter to the Duchenne Community: Update on IGNITE DMD Clinical Trial


- Full Clinical Hold Letter from the FDA Received; Company to Respond in the Coming Weeks -

- Manufacturing-related Partial Clinical Hold on the High Dose of SGT-001 Resolved -


Solid Biosciences Inc. (NASDAQ:SLDB) today reported financial results for the full year ended December 31, 2017 and provided a corporate update.


Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent.


Solid Biosciences Inc. (NASDAQ:SLDB) today announced it has received notification from the U.S. Food and Drug Administration ( FDA ) that IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.


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