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                                            

Related news


Solid Biosciences is proud to join the global rare disease community again this year to observe Rare Disease Day


Today we are excited to announce the initiation of our first clinical trial for SGT-001.


Phase I/II adaptive clinical trial to evaluate safety and efficacy in ambulatory and non-ambulatory children and adolescents.


Synpromics Ltd, the leader in gene control, today announces a new research partnership with Solid Biosciences, LLC. 


New data from the SGT-001 preclinical program was published today in Molecular Therapy – Methods and Clinical Development


We are getting ready to initiate a clinical study for our gene transfer candidate, SGT-001, later this year.


Solid Biosciences is pleased to announce additional research grant from our Australia-based partner Save Our Sons and an initial research grant from Ireland-based Join Our Boys.

  • Jennifer Ziolkowski, CPA, joins as Chief Financial Officer.
  • Carl Morris, Ph.D., promoted to Chief Scientific Officer.
  • Joel Schneider, Ph.D., promoted to Chief Technology Officer, Head of Exploratory R&D.

Company to Use Proceeds to Advance Programs for Duchenne Muscular Dystrophy.


Solid Biosciences is proud to join the global Rare Disease community to recognize Rare Disease Day 2017.


More information about Solid?

Please subscribe to receive our latest news