Guest Post: ALS an Orphan Disease? Not in Massachusetts.
The 8th Annual ALS TDI Leadership Summit is just around the corner so Robert Goldstein, the Vice President of Communications & Public Affairs of ALS TDI, wrote in to share a bit about ALS research breakthroughs and the upcoming event with our readers:
Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s disease) is universally fatal. It is considered an orphan disease. However, every 90 minutes, someone is diagnosed. In Massachusetts alone, there are 130 new cases a year. Without an effective treatment, half will not survive more than a couple of years on average.
There are some well-known ALS patients in our state, like former Governor Paul Cellucci, and ALS advocates, like former Red Sox pitchers Curt Shilling and Mike Timlin. However, the unique innovative and entrepreneurial mindset here in Massachusetts has enabled some of the most important advances in ALS research. We don’t have a cure yet, but we are making progress. In fact Massachusetts-based researchers, non-profits, and companies have led the way since the early 90s:
That is just the short list. Regardless whether you are a scientist, in BD, a venture capitalist, philanthropist or clinician seeing patients daily at one of our state’s world-class hospitals, you likely have three questions on your mind – What do I need to know? What’s going to happen next? How close are we?
These and other questions will be answered directly, when a large group of innovators and decision-makers gather for the 8th Annual ALS TDI Leadership Summit in Boston on November 1st. This event is hosted by TDI and is completely free and open to the public.
Here are just a few highlights to look forward to:
- Steve Perrin, CEO/CSO of TDI, will reveal data on nine previously unpublished compound screens;
- Mark Namchuk of Vertex will discuss the unique partnership with the Cystic Fibrosis Foundation to develop the first treatment for that disease in decades;
- Robert Brown will expound on his recent work that helped identify the 19th genetic association for ALS (EPHA4);
- And the Wall Street Journal’s Amy Marcus will challenge all to answer the role of the patient and responsibility of the groups each panelist represents.
Click here to register for the ALS TDI Leadership Summit.
Robert Goldstein is the Vice President of Communications & Public Affairs at the ALS Therapy Development Institute. He is responsible for the execution of a strategic communication plan for ALS TDI and continuing to increase its profile as an agenda-setter in the field of neurodegeneration.
Visit ALS Therapy Development Institute’s website.
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Posted on October 16, 2012, in Guest Bloggers and tagged ALS, ALS TDI, ALS Therapy Development Institute, Lou Gehrig’s disease, Massachusetts, Robert Goldstein. Bookmark the permalink. Leave a Comment.