After decades of limited progress in amyotrophic lateral sclerosis (ALS) research, a relentlessly progressive disease that affects approximately 30,000 patients in the United States, an investigative therapy may offer a spark of hope and a major step forward. Anticipation for new breakthroughs in the complex disease research has been building for the patients and families who have little time to wait.
In the past, an ALS diagnosis often meant a startling decrease in length of life. Even with at least 100 drug trials being conducted in the past 30 years, only two drugs were eventually approved for patient use, but patients still only live about three years after being diagnosed, on average.
ALS often begins with symptoms such as unexplained weakness, vocal changes or shortness of breath. These develop into a spreading paralysis which results in people being unable to walk, eat, talk, move their arms or breathe. On average it affects people in their 50s, but cases can occur in people’s 20s and 30s as well, albeit rarely. The lifetime risk of developing ALS is approximately 1 in 400.
The good news is that research is finding new treatments help to slow this disease and improve overall quality of life. A new investigational treatment has shown an ability for patients to retain function and independence longer, and the findings from that study are now being published in a leading medical journal. ALS as a disease has been notoriously challenging for new drug development, and this represents a historic step forward.
On Thursday, September 17, Dr. Sabrina Paganoni, Assistant Professor at Harvard Medical School, Justin Klee and Joshua Cohen, Co-Founders and Co-CEOs of Amylyx, will talk about the latest published research & clinical trial results that may help those with ALS improve their overall quality of life.
When you book a window, we’ll provide you with guest bio & suggested questions. Interview is courtesy: Amylyx