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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PhRMA Report: Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View

For Immediate Release:  January 17, 2013

Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released a report entitled “Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View.”  The report was conducted by researchers at the Analysis Group, and examines the drug pipeline to illustrate the type of research conducted by biopharmaceutical companies.

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Congressional bickering is deeply damaging to biomedical research

The inability of Congress to pass a budget is damaging the NIH, the core of biomedical research in the U.S, which has cut or delayed funding for most new top projects, biomedical researcher Steven Salzberg writes in Forbes. He says this has left scientists and researchers looking for work, possibly in the private sector, and he calls on members of Congress to set aside their fighting and just pass the NIH budget without cuts. 

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From FDA Matters: Orphan Drugs at 30: Will Success Become Too Expensive?

The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact.  Amidst the celebrations, a reporter asked me a provocative question: can we afford more orphan drugs costing hundreds of thousands of dollars per year? FDA Matters answered “yes.”

From The Alliance for a Stronger FDA: Points to Keep in Mind for Next 8 Weeks

The 8-week reprieve from sequestration has benefited all federal discretionary programs. “Live to fight another day” is not to be taken for granted.  Sequestration still, however, looms as an existential threat to the mission of the FDA. If it occurs — as presently scheduled, on March 1 — we estimate the agency will have to generate about $250 to $270 million in savings over 7 months.

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