Searching for Success

Searching for Success

02.10.11 | By Kate Connors

Yesterday, our friends at ALS Advocacy Tweeted about the Chart Pack: "Need some #ALS success charts in the next edition!"

They can rest assured that America's biopharmaceutical research companies are working to create success stories for ALS (also known as Lou Gehrig's Diseases) and many other rare conditions.

ALS is a tragic disease, one that affects up to 30,000 patients in America, according to the ALS Association. Beyond those who are diagnosed, the loved ones and caregivers affected are innumerable.

Until recently, there was no known therapy that affected the progression of the disease, rather than the symptoms. Finally, in 1995 came a breakthrough, when the first - and only - medicine was approved to treat ALS.

However, it's not a cure, and challenges remain for Americans who battle the disease daily. That's why research on ALS is ongoing, as it is for many rare diseases.

Also known as "orphan diseases" (the medicines to treat them are known as "orphan drugs"), they are defined as affecting fewer than 200,000 Americans; many are much, much smaller, affecting only a few thousand patients.

Because of their size, these rare diseases don't get a lot of public attention, but you can be sure that to the patients and loves ones affected, they're a number one priority. Hopefully February 28 will raise awareness as we recognize national Rare Disease Day. This month, PhRMA will be releasing our report on the number of orphan drugs in development to treat rare diseases.

You'll have to wait until then to see the full results of the study, but here's a teaser for our friends at ALS Advocacy, and the patients they represent: the report shows seven new medicines being studied to treat ALS. Perhaps one of them will be a success story in the future.

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