FAST Act – Speeding RARE Disease Therapies
Last week over 80 RARE Disease advocates lobbied on Capitol Hill to enable better access to Accelerated Approval of therapies. We discussed H.R. 3737 “Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA)” during our day on the hill. Today, an additional piece of bipartisan legislation, the FAST Act (HR 4132), was introduced in the House. Incorporating [...]
How A Bill Becomes a Law … It’s Complicated
We are often asked why we can’t quickly change the laws related to RARE disease therapy approval. The answer is that not only are politics always involved, it’s that the process is very complicated. This cartoon depicts what often happens in this process … what is needed seems very clear to us, but what is [...]
Take Action: Tell Congress Rare Diseases Patients Deserve Treatments
Tomorrow is a day of celebration, but TODAY is a day of ACTION. More than 70 patients advocates are on the Hill today meeting with Members of Congress. Please join them in the Lobby Day by taking 10 minutes to call your Representatives and ask them to support efforts to spur lifesaving treatments for rare [...]
RARE Disease Drug Development … it’s complicated!
The development of any drug is a very complicated process … for RARE diseases the task is especially difficult due to the limited patient populations. Typically the disease is less well understood and there are fewer patients to access for testing to prove the drug works. We’re hoping this simplified overview of the process will [...]
RARE But Aware: What You Should Know About World Rare Disease Day
RARE Disease Day Blogs and Amylin Pharmaceuticals Since 2010, Amylin Pharmaceuticals, Inc.’s blog, Building Blocks, has continued to bring science, and dedication to changing lives through communication. In Building Blocks - Amylin’s Blog explores top discussions in healthcare, policy, patient commitment and latest scientific advancements in managing diabetes and obesity. Recently, our very own Nicole Boice, [...]
February 29 – A Celebration of Unity
The Month of February During the month of February, much of the American population will be celebrating historical events, special interest groups and campaigns. Some of these include Black History, American History, Children’s Dental Health, and American Heart Month. Internationally recognized events in February include National Embroidery month (my favorite), Wild Bird Feeding, Cherry and [...]
A Life with PKU (Phenylketonuria)
‘THIS IS MY NORMAL’ Kevin Alexander is a normal guy. He likes to play electric guitar, video games, and write music. He enjoys listening to movie sound tracks, playing in a band and is a professional Videographer. What most people would not realize about Kevin is he is living with Phenylketonuria (PKU) – [...]
RARE DISEASE DAY EVENTS
Looking for a way to participate in Rare Disease Day Events? Don’t miss these Rare Disease Day events in DC! Monday, February 27, 2012 RDLA Luncheon Meeting & Conference Call (Host: Rare Disease Legislative Advocates) 12:30 – 2:00p.m. at 750 9th Street NW, Suite 750 Washington, DC 2nd Annual RDLA Cocktail Reception and [...]
RARE Disease Lobby Day – February 28 Washington, DC
RDLA is partnering with the EveryLife Foundation for Rare Diseases, RARE Project, Sarcoma Foundation of America, Darlington House and ViroPharma to ensure that rare disease advocates are present on Capitol Hill during Rare Disease Week! Join us and make sure Congress hears your voice! Lobby Training Breakfast 7:30a.m. – 9:30a.m. Darlington House 1610 20th ST NW Washington [...]
PATIENT STORIES OF HOPE AND INSPIRATION
‘MEET THE SPOONER FAMILY!’ Rick and Cristyn Spooner of Rancho Santa Margarita have fostered a dynamic husband and wife team both as business partners and in raising three beautiful children. Despite achieving success in operating their loan company, the Spooners have faced greater challenges and discovered even greater meaning in their lives through the caring [...]