Crohn’s & Colitis Caucus Briefing: Patient Access to Affordable Treatments

May 23rd • Noon • Lunch Provided
Rayburn House Office Building 2168

“Patient Access to Affordable Treatments” will provide attendees with an update on the Congressional Crohn’s & Colitis Caucus, as well as a dialogue with our panel of experts from the scientific community on patient access to drug treatments.

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Shire Launches Report that Quantifies the Health, Psycho-social and Economic Impact of Rare Diseases

Lexington, Massachusetts, US – April 9, 2013 – Shire plc (LSE: SHP, NASDAQ: SHPG) today launched a Rare Disease Impact Report, which uncovers the health, psycho-social, and economic impact of rare diseases on patient and medical communities in the United States (US) and United Kingdom (UK). The report, developed in collaboration with an external advisory board of thought leaders in the medical, advocacy, health policy and health economics fields, will be distributed at the World Orphan Drug Congress in Washington, DC (April 9-11) and is available for immediate download at www.rarediseaseimpact.com.

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April 16th Rare Disease Congressional Caucus Briefing

Rare Disease Legislative Advocates and Novartis, in coordination with Rare Disease Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY)  will host a briefing on “Medical Innovation for Rare Diseases: Challenges that Our Country Must Address.”  Tuesday, April 16, 2013 from 12:00 PM – 1:00 PM in Rayburn B-318

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BRAIN Initiative Challenges Researchers to Unlock Mysteries of Human Mind

From the White House Blog:

Today at the White House, President Obama unveiled the “BRAIN” Initiative—a bold new research effort to revolutionize our understanding of the human mind and uncover new ways to treat, prevent, and cure brain disorders like Alzheimer’s, schizophrenia, autism, epilepsy, and traumatic brain injury.

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April 17th: Newborn Screening Lunch Briefing: 50 Years of Saving Babies’ Lives

Wednesday, April 17, 2013

LUNCH BRIEFING: 12:00 – 1:30pm B-340, Rayburn House Office Building

Did you know that each of the more than 4 million infants born in the United States this year will undergo newborn screening? Newborn screening is a vital state public health activity that relies on research and science based guidance from federal agencies. Screening detects conditions in newborns that, if left untreated, can cause severe lifelong disability, developmental delays or even death. Approximately 1 in every 300 newborns in the United States has a condition that can be detected through newborn screening.  This year marks the 50th anniversary of newborn screening in the United States.

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Rare Disease Caucus Chairs Recognize Rare Disease Day

(Washington, D.C.) – Today, Reps. Joe Crowley (NY-14) and Leonard Lance (NJ-7), co-chairs of the Congressional Rare Disease Caucus, issued the following statement in recognition of Rare Disease Day: 

“Today, we join the millions of patients, their families, and the global health community in observing Rare Disease Day.

“For the 30 million Americans suffering from a rare disease, half of whom are children, medical advancements can’t come soon enough. Most rare diseases have no current treatment available, and many are not even studied by researchers.

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Webinar: The 113th Congress and Medical Research Funding: A Perfect Storm Approaching?

This is no garden-variety budget year in Washington – a tsunami of budget and fiscal issues threaten federal science programs. While Congress has pushed the date of the “sequester” off until the beginning of March, delaying the prospect of automatic 8.2 percent cuts in the budgets of NIH, FDA, and other agencies, it is not entirely off the table. Current funding for these programs expires in March, the fiscal year 2014 budgets are being drafted, and there will likely be a fight over the debt ceiling.  This Webinar will tell you what you need to know about what could happen on Capitol Hill, when, and who the important players are, including some new faces.

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Senator Brown wants more pediatric-research funding: Bill would shift more federal money to kids’ illnesses

THE COLUMBUS DISPATCH Tuesday January 29, 2013 8:24 AM
By  Joshua Jamerson 

When Madison Reed was 7 months old, doctors told her parents she wouldn’t live past 2. “No parents should have to hear those words,” Annette Reed said. But unconventional methods helped Madison, who had type-1 spinal muscular atrophy, a rare disease impairing her motor functions. Now the Dublin resident is 15 years old.

The Reeds stood behind Sen. Sherrod Brown yesterday at Nationwide Children’s Hospital as the Ohio Democrat used their story to illustrate the need to increase funding for pediatric research.

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RDLA’s 3rd Annual Rare Disease Cocktail Reception & Movie Screening

Join RDLA for our 3rd Annual Rare Disease Cocktail Reception & Movie Screening to kick-off Rare Disease Day (week) in Washington DC

Monday, February 25, 2013
5:00pm Cocktail Reception * 6:30 Movie Screening * 8:00 Panel Discussion
Carnegie Institution of Science, 1530 P Street Northwest, Washington DC

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Feb. 6 Briefing: The Blueprint of Medical Research: How New Medicines Get from the Lab to the Patient

Improving and accelerating the R&D path requires all stakeholders in the medical research ecosystem to work together. Co-hosted by FasterCures and Friends of Cancer Research, this briefing is designed to educate members of Congress, legislative staff and advocates about the vital role federal agencies and the medical research sector play in advancing medical progress, and to emphasize the importance of prioritizing this work.

Wednesday, February 6, 2013, 12:00pm – 1:30pm
Dirksen Senate Office Building, Room 106, Washington, DC

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