The ALS Association

ALS Ice Bucket Challenge Progress

The ALS Association joins the Pharmaceutical Research and Manufacturers of America (PhRMA) in releasing new report

May 9, 2016

Today, The ALS Association joins the Pharmaceutical Research and Manufacturers of America (PhRMA) in releasing a new report, “Medicines in Development for Rare Diseases,” which finds that America’s biopharmaceutical research companies are currently developing more than 560 medicines for patients with rare diseases, including amyotrophic lateral sclerosis (ALS).

There are about 7,000 known rare diseases, or those that affect fewer than 200,000 people in the United States every year. Oftentimes, development of treatments for rare diseases poses many challenges. According to this new report, researchers are leveraging new technologies to advance drug development.

“It is collaborative efforts between biopharmaceutical researchers, patients and disease groups like ours that are driving the search for new treatments for ALS,” said Barbara Newhouse, president and chief executive officer of The ALS Association. “As we learn more and more about this debilitating disease, the prospect of a cure is becoming a reality – giving patients and their families new hope.”

Currently, there are 38 drugs in development for neurological disorders, including ALS. New therapies under development, like antisense technology against SOD1, are a step toward helping patients and their families manage ALS.

ALS Advocate Lorri Carey, who is living with ALS, is highlighted in the report along with her work to help Congress establish the National ALS Registry and create patient-focused drug development guidance to accelerate therapies to slow or stop ALS.

“I’m one of the lucky ones,” she says. “I was hoping to live long enough to see my sons graduate from high school. As it turns out, I’ve been blessed to have seen them graduate from high school and college. With my sons and husband, together, we’re chasing the dream: a world without ALS.” Click here to read Lorri's full blog post.

Read the report.

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