About Us
Sharon Hesterlee, PhD
Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association, can address:
(1) MDA’s history with funding neuromuscular disease research; (2) how MDA’s care for the neuromuscular disease community changed during the COVID-19 pandemic; (3) hope for the future in terms of research and care for patients living with a neuromuscular disease.
Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association Bio:
Sharon Hesterlee, Ph.D., is the Chief Research Officer at the Muscular Dystrophy Association. Prior to rejoining MDA, Dr. Hesterlee served as the Executive Vice President for Portfolio Development and head of neuromuscular programs at leading adeno-associated virus (AAV) gene therapy company Asklepios Biopharmaceuticals, Inc. (Askbio). Before joining Askbio/Lion she served as Director, Gene Therapy at Pfizer Inc.’s Rare Disease Research Unit, where she led three of Pfizer’s internal gene therapy programs following the acquisition of Bamboo Therapeutics in 2016. In the 15 years before transitioning to industry-focused roles, Dr. Hesterlee led research and strategy for several leading nonprofit organizations, having served as Chief Science Officer for the Myotonic Dystrophy Foundation, Vice President of Research for Parent Project Muscular Dystrophy and Scientific Director of the Association for Frontotemporal Degeneration. During her initial work with MDA, she served as Senior Vice President and Executive Director of MDA Venture Philanthropy and Vice President for Translational Research, among other positions.
Quotes from Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association:
“MDA originally funded the discovery of dystrophin, the gene that is responsible for Duchenne muscular dystrophy when it has mutations. That was a big deal: That was the first gene ever identified through positional cloning, which was a new technique at the time. We’re at another crossroads where there’s incredible progress being made, and I think we can see where MDA funds can make a big difference. What is our next big moonshot? Let’s fund it and make that happen. I will say it will probably be in the genetic medicine space. We’ve made a lot of progress. Let’s keep that going.” – MDA
“DMD is an area that has received a lot of research funding from MDA. Over the years, we’ve put approximately $221 million into the research that underlies this form of muscular dystrophy. In the 1980s, we spearheaded the effort that identified the gene, that when defective, is responsible for the disease. It was the first disease-causing mutation in a gene identified by positional cloning, which was a completely new technique at the time.” – Developments in Specialty Pharmacy
“Both Sarepta and Pfizer have collected some promising functional data. It is very likely that one or both of these gene therapies could be approved. This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation. It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.” – BioSpace
Expertise
- MDA’s history with funding for neuromuscular disease research
- Care for the neuromuscular disease community
- Gene therapy
- Emerging therapies for neuromuscular disease
- Regulatory challenges in neuromuscular disease
- Clinical trials
- MDA’s MOVR Data Hub™
Social Media
Sharon Hesterlee in the Media
- MDA Chief Research Officer Dr. Sharon Hesterlee Shares MDA’s History
- MDA Chief Research Officer Dr. Sharon Hesterlee Discusses Remote Care
- MDA Chief Research Officer Dr. Sharon Hesterlee Talks Future Research
Contact
- Email: press@mdausa.org
