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Feb 25
Jeff Boutelle, Head, U.S. Rare Disease Strategy at UCB
Recognizing Rare Disease Day Every Day – The Importance of Addressing Unmet Patient Needs

At UCB, we work 365-days-a-year to find new ways to deliver solutions, peace of mind, and opportunities to people living with severe diseases because everyone deserves to live the best life they can. This Rare Disease Day, I want to share a bit about why this mission isn’t just professional for me, it is personal. 

Two weeks after starting in my role as Commercial Lead for the Rare Disease Organization at UCB, I discovered that my favorite uncle, Uncle Ed, had been diagnosed with generalized myasthenia gravis (gMG). Myasthenia gravis (MG)– a chronic, autoimmune neuromuscular disease, where the body’s immune system mistakenly attacks the connections between nerves and muscles – may be hard to diagnose. MG leaves patients with unpredictable symptoms like fatigue, muscle weakness, and difficulty breathing and swallowing.1 Generalized myasthenia gravis is a more severe form of the disease that not only alludes early diagnosis but is often hard to treat. Between 36,000-60,000 people in the U.S. are estimated to have MG.2 Uncle Ed first knew something was off about six years ago, at age 69, when his eyelids started to droop and his speech slurred. He started to choke on his food, bite his tongue and cheeks, and lose strength in his arms and legs.  

After receiving his diagnosis, Uncle Ed visited a Veterans Affairs (VA) hospital, as he is a Vietnam War veteran and retired as a three-stripe Sergeant of the U.S. Army 101st Airborne Division. The VA started Uncle Ed on steroids, but relief didn’t last long. They then followed with infusions of antibodies which are now administered over a period of four days and have lasting effects of three to four months before the onset of his symptoms. If he is unable to get his treatments in a timely manner, it negatively impacts his quality of life and those around him. One of my cousins moved in with him to keep an eye on him. When his symptoms exacerbate, he can get confused and slur his speech – for Uncle Ed and his family, there isn’t a day that passes where his MG isn’t top of mind.

Because of this and other experiences MG patients have shared, I am proud UCB is undergoing efforts to investigate novel therapies for rare disease including MG. UCB’s origins in rare disease come from a UCB scientist, who was looking for additional treatment options. This same need for additional therapies continues to remain an important issue for Uncle Ed.

Currently, he needs to be driven an hour and a half each way to receive his treatment, a life-upending experience shared by many living with MG and other rare diseases. 

As a result, UCB remains committed to patient value and changing the day-to-day experience for people living with MG. We are living out this commitment in a number of ways, including:


  • Developing a promising pipeline of differentiated therapy options that have completed Phase 3 clinical trials and look forward to submitting them for FDA review later this year; 
  • Focusing on patient-centered research, comprehensive disease and scientific education, and advocacy efforts;
  • And analyzing public policy opportunities that address current challenges facing patients including accessibility, affordability, and telehealth. But we can’t stop there. 


As we look into the future of MG care and how UCB can help improve people’s lives, we must ensure that patients, like my Uncle Ed and others, can get the diagnosis and treatment they need in a timely manner by making it simple and easy for physicians to prescribe medicines and for patients to get and stay on effective treatments. 

“I have the support I need through the VA, but I think it is important to know how serious these symptoms can be. Not everyone is as lucky as I am to have the VA take care of things in a timely manner,” said Ed Boutelle. “It is important that when people with MG start to show symptoms, they can get the treatment they need so they don't need to suffer with this condition.” 

Please join us as we raise awareness of rare diseases like MG on this Rare Disease Day. It is more than just a day. It recognizes a movement focused on finding patient-centric solutions and developing stronger policies to tackle unmet patient healthcare needs. 

Pictured: Ed Boutelle, "Uncle Ed"

Pictured: Courtney Peyer, Ed Boutelle, Jay Boutelle, and Hannah Boutelle