51: Episode 50 - PJ's Protocol: A Lifesaving Procedure Fueled by Love

In this Quest Podcast episode, we chat with PJ’s father, Brian Nicholoff, Director of Patient Access at the Little Hercules Foundation, Amy Aikens and emeritus professor of Pediatrics and Neurology, Dr. Jerry Mendell. Brian has been an advocate for Duchenne Muscular Dystrophy for nearly 40 years and Amy advocates for treatment access concerns and works directly with individuals and families to address individual barriers. Dr. Mendell, in his over 50-year career, has devoted efforts to understand Duchenne muscular dystrophy and pioneered the standard of care that led to FDA approval for deflazacort and vamorolone. Their goal is to advocate for the creation successful treatments and eventually a cure for those effected by neuromuscular diseases. They join us to share their experiences, expertise, and advice.

Transcript

Guests: 

Brian Nicholoff has been married to his wife, Barbara, for 46 years and is a life member of the Professional Golfers Association of America. They are parents to Phillip James (PJ) 1982 and Justin Alan 1987. Brian is retired and enjoys spending his time with his wife, Justin and his friends. He especially loves traveling, music and writing poetry. He has been an advocate for Duchenne Muscular Dystrophy for nearly 40 years.

PJ passed in 2013 and the Protocol in his name was established in March of 2015. It is now in several languages and referred to by countless families and physicians in many parts of the world.

Connect with Brian: 

• LinkedIn: https://www.linkedin.com/in/brian-nicholoff-08693520/

Amy Aikins has been led to her current professional career path from her experience as a mother to a young man with Duchenne Muscular Dystrophy. She has utilized her personal experience navigating systems and overcoming barriers for her son to assist others. 

Amy has spent her professional life working in various capacities within organizations focused on serving individuals with disabilities and has a passion for helping people obtain access to the things that they need. In her current role as Director of Patient Access at the Little Hercules Foundation, Amy tackles treatment access concerns through extensive engagement with a variety of stakeholders, as well as involvement in various groups and projects focused on access issues within the rare disease community. In addition, she works directly with individuals and families to address individual barriers. 

Connect with Amy:

• Email: [e-mail] 
• LinkedIn: https://www.linkedin.com/in/amy-aikins-bcpa-75a3b613a/

Dr. Jerry R Mendell attended medical school at UT Southwestern Medical Center and Residency at Columbia University. Post graduate fellowship at NIH and first employment provided contact with first patient with Duchenne, inspiring decades of work as a translational clinician and researcher. Most of his achievements were done at Nationwide Children’s Hospital where he was recruited to establish a Neuromuscular Center. Over a 50-year career, he devoted efforts to understand Duchenne muscular dystrophy and ways to intervene. He was the lead author introducing prednisone for DMD treatment published in the NEJM in 1989. The importance is validated in over 30 publications. It is standard of care and led to FDA approval for deflazacort and vamorolone.

He is an emeritus professor of Pediatrics and Neurology at The Ohio State University and Nationwide Children's Hospital where he held Curran-Peters Endowed Chair in Pediatric Research. He is now a Senior Advisor at Sarepta Therapeutics. In 2024, he was named to the inaugural TIME100 Health most influential people in Health pioneering a new age of treatment for genetic diseases. Dr. Mendell was the 2024 recipient of the King Faisal Prize Laureate in Medicine. In 2020, he was elected to the National Academy of Medicine and was the first recipient of the American Society for Gene and Cell Therapy’s "Jerry Mendell Translational Medicine Award" now given to other scientists in his name each year at the annual meeting. He has authored 420 articles and written 3 books about muscle and nerve disorders and gene therapy. The Center for Gene Therapy at Nationwide Children’s has now been named in his honor, the "Jerry Mendell Center for Gene Therapy".

Connect with Dr. Mendell: 

• Instagram: https://www.instagram.c