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Orphan Diseases

Late Onset Tay Sachs Clinical Trial

Alexis Buryk | Posted 10.05.2015 | Impact
Alexis Buryk

I'm incredibly impressed with the work being done by the NIH. And also feel I have to do more. I fully intend to contact Genzyme and find out more about the drug. I don't know how proprietary it is or if it is being distributed in any other country. I need to know more to help my children.

Efforts Initiated to Conduct First FDA Phase I Trial for ALS

April Rudin | Posted 07.30.2015 | Impact
April Rudin

Although designated an "orphan disease" because it affects less than 200,000 Americans, Amyotrophic Lateral Sclerosis (ALS) saw millions of benefactors stand tall last year to douse themselves with ice water in support of a cure. The numbers were staggering.

How to Get Researchers to Notice an Ultra-Rare Disease

Idelle Davidson | Posted 03.01.2015 | Healthy Living
Idelle Davidson

It doesn't help that Erdheim-Chester is notoriously difficult to diagnose and therefore, say researchers, most likely under- or mis-diagnosed. Only when there's a mass or lesion to study will pathologists look for identifying markers on cells -- if they even suspect ECD.

Celebrating World Lupus Day: Gaining Ground With Effective Policy

Chester Aldridge | Posted 07.10.2014 | Impact
Chester Aldridge

Today celebrates a decade-long movement to gain public and private interest to increase financial support, resources and assistance for the chronic, autoimmune disease that affects an estimated 2 million people in the United States alone.

A Peloton to Stave Off the Loneliness of Orphan Cancer

Rick Kaitz | Posted 09.24.2013 | Impact
Rick Kaitz

After what was described to us as "routine" surgery to remove a fibroid, my wife, Erica, was diagnosed with Myxoid, uterine leiomyosarcoma (LMS). That was the first shock. The second? The cancer is so rare, each year fewer than 100 people in the country receive such a diagnosis.

Finding a Home for Orphan Diseases

Daniel P. Malito | Posted 03.24.2012 | Healthy Living
Daniel P. Malito

Recently, I spoke with someone who opened my eyes to an entire class of diseases for which there are few to no medications available, and almost no plans to create any remedies in the near future. These ailments are called "rare" or "orphan" diseases.