Skip to main content

In the tradition of heroes

When I tell you that someday I hope I have the time and resources to write a book about the rare disease community and some of the many wonderful and inspiring stories out there, I'm not kidding. Here's one for which we are all grateful. I took this announcement from the Genetic Alliance newsletter.

2007 Art of Advocacy Award Winner

The Art of Advocacy Award pays tribute to a visionary grassroots leader who is harnessing his or her knowledge and experience to improve the quality of research, healthcare, information and support services for a specific condition or for a coalition of grassroots organizations.

Attend the Awards Banquet. Register
here.

Abbey Meyers, National Organization for Rare DisordersTwenty-five years ago,” describes an industry representative, “this mother from Connecticut took the passion that only a loving and concerned parent can have and turned it into a vision that energized other parents of children with unmet medical needs stemming from rare diseases, that motivated physicians and other healthcare providers seeing these unusual and oft-undiagnosed disorders, and that enlightened elected public officials.” In 1983, Abbey Meyers founded the National Organization for Rare Disorders (NORD), a federation of voluntary health organizations dedicated to helping people with rare “orphan” diseases, which is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. Leading the way through NORD, Ms. Meyers is considered to be the primary consumer advocate responsible for the passage of the landmark Orphan Drug Act of 1983.The president of a rare disease advocacy organization states, “All of the tens of thousands of Americans with rare diseases who have benefited from the medicines developed for them under the Orphan Drug Act can be grateful that Abbey has the mind, heart, and voice to have been the ultimate advocate for them.”Genetic Alliance is proud to honor Ms. Meyers with the Art of Advocacy Award for her vision, dedication, and experience in moving the rare disease community forward. Her 25 years of tireless work has touched the lives of countless patients and families worldwide. Thank you, Ms. Meyers for everything you have done and will continue to do.

Comments

Popular posts from this blog

Ratner's Cheesecake

Here's another recipe from Toby! Thanks Toby......and I'll get the others posted soon! Ratner's Cheesecake and plain cookies Ratner's was a Jewish dairy restaurant in the lower East Side of Manhattan. This recipe, from my disintegrating, no longer in print Ratner's cookbook, is the closest I've ever gotten to reproducing the rich, heavy cheesecake my mother made when I was a kid. It's worth the time it takes to prepare and every last calorie. Dough Can be prepared in advance. Makes enough for two cakes. Can be frozen or used to make cookies – see recipe below. 1 cup sugar 1 teaspoon vanilla extract 1 cup shortening 1 teaspoon lemon extract 1 cup butter 2 eggs 3 cups sifted cake flour ½ teaspoon salt 2 cups all purpose flour 1 teaspoon baking powder 1. In a bowl, combine all ingredients with hands. Refrigerate 3 -4 hours, or preferably overnight. Filling (for

The next generation with Hermansky-Pudlak Syndrome

I'm so behind on posting about the trip to Puerto Rico. Since the episode of Mystery Diagnosis on Hermansky-Pudlak Syndrome ran right after we got home, it's been a little busy. These, however, are my favorite pictures from Puerto Rico. I know, not pretty senery etc - but these little guys and gals inspire me. They are the next generation of folks with HPS, and if we keep up the hard work, they will live better lives because of it. They motivate me.

Some good news about Pirfenidone

Below is a press release from Intermune, the company that makes Pirfenidone. They have essentially reviewed the various clinical trials going on, and decided that Pirfenidone is safe and well tolerated. That would pretty much go along with what we've observed in the HPS community as well. We have a few folks that have been on the drug since the late 90s and continue to do well. Of course, as a journalist, I do have to say consider the source - but at the same time, as someone in a Pirfenidone trial, it's good to know. Results of Comprehensive Safety Analysis of pirfenidone In IPF Patients Presented At European Respiratory Meeting - Analysis shows safety and tolerability of pirfenidone across four clinical trials - VIENNA, Sept. 14 /PRNewswire-FirstCall/ -- InterMune, Inc. (Nasdaq: ITMN ) today announced that the results of a comprehensive review of safety data from four clinical studies were presented at the 2009 European Respiratory Society Annual Congress in Vienna, Austria