Living with Duchenne muscular dystrophy (DMD)? If you aren’t familiar with Solid Biosciences, you should be!

Here at Patient Worthy, we give credit when credit is due. We root for new treatment options and keep our eyes on the horizon for the latest rare disease news.There are a few organizations that deserve some serious kudos!

Solid Biosciences exists to find a cure for patients living with Duchenne muscular dystrophy. They partner with several organizations, labs, and groups all to develop a “treatment that extends life” for those with the fatal DMD, and they aren’t afraid to open their pockets to fund promising research.

The Gainesville Sun reported that The University of Florida’s Powell Gene Therapy Center is conducting patient worthy research toward treatments for DMD on behalf of Solid GT, a subsidiary of Solid Biosciences. They put up $2million to the Powell Gene Therapy Center to continue DMD lab research to date. You must be wondering about the specific kind of research that elicits such a click bait headline:

So what do mice and golden retrievers have to do with DMD?

University of FL Center Director Dr. Barry Byrne says that UF has already produced a “shortened but functional version of dystrophin” – a protein group that boosts muscle strength and protects muscles from injury. UF has delivered this dystrophin to mice and golden retrievers and Dr. Byrne says Solid GT’s funding will continue the very promising, animal research with the goal of getting the “OK” from the FDA to start clinical trials.

“Yikes!” some of you may say. “I don’t believe in animal testing – it is super expensive and it’s just not right to intentionally harm another living being.”

Others may think, “Well how else do you think we came to have HIV and cancer drugs, antibiotics, or insulin?” or “Well, it helps determine the safety of pipeline drugs.”

We want to hear from you. Let’s talk the Pros and Cons of animal testing. How does the painfully obvious gap between development of drugs for common versus rare diseases influence your stance?

Patient Worthy is going to keep an eye on Solid GT and all of their DMD efforts. We will be sure to keep our readers up to date with the latest on Duchenne muscular dystrophy treatment research. Don’t forget to let us know your thoughts in the comments below.

For more of Solid GT’s latest news:

• In September of 2015, Debiopharm International SA and Solid Biosciences, LLC, announced their collaborative exploration of the use of Alisporivir (Debio 025) in muscular dystrophy. You can check out the full Press Release here.
• Back in November of 2015, they raised $42.5 Million to further the development of its gene therapy platform. You can check out the full Press Release here.