DUCHENNE NOW Competitors

If you’re seeking to start or advance your career with a purpose, Muscular Dystrophy Association is an employer that will enable you to thrive. The Muscular Dystrophy Association is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. Our mission is to empower the people we serve to live longer, more independent lives. #MDA #MuscularDystrophy #ALS #neuromuscuar. Apply now.

Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com. Solid is headquartered in Charlestown, MA, USA.

Empowering the Next Generation Rare Disease Advocate. Global Genes and RARE-X officially merged into one organization December 2022 under the name Global Genes. Both organizations have been working toward the goal of empowering the next generation advocate both through their individual initiatives and their strategic partnership announced in 2021. RARE-X was born out of Global Genes after discussions with advocacy leaders, board members, partners, patients, and care partners, addressing a significant gap in access to tools, technology, and infrastructure. Hope. It’s in our genes.™