Entries by Andrew Hughey

National PKU Alliance Asks Advocates to Contact Representatives to Cosponsor the Medical Nutrition Equity Act, H.R. 2105

The National PKU Alliance is asking advocates to contact their Representatives to cosponsor the Medical Nutrition Equity Act. There are currently 16,500 Americans living with Phenylketonuria (commonly known as PKU). PKU is a rare, inherited metabolic disorder that causes inadequate growth, developmental delays, intellectual disability, and behavioral disorders without treatment. The first line of therapy […]

The EveryLife Foundation Asks Advocates to Contact Representatives to Cosponsor the Reauthorization of the Newborn Screenings Saves Lives Act

The EveryLife Foundation for Rare Diseases is asking advocates to contact their Representatives to cosponsor the Newborn Screening Saves Lives Act. Legislation to reauthorize the Newborn Screening Saves Lives Act was introduced in the House of Representatives by Representative Lucille Roybal-Allard on May 2, 2019. Introduction in the Senate is expected soon. H.R. 2507, the […]

Patient Focused Drug Development

Parent Project Muscular Dystrophy (PPMD) is taking the lead on strengthening the voice of the patient throughout the drug and larger medical product development process. They are doing this by urging congress to add  transparency requirements in upcoming 21st Century Cures Legislation. PPMD has written a sign-on letter to urge that a provision known as the […]

2014 RDLA’s Legislative Scorecard

Rare Disease Legislative Advocates compiled a congressional scorecard to educate advocates on how their Representative has voted on issues affecting the rare disease community.  Each Representative’s score is based on membership in the Rare Disease Congressional Caucus as well as action taken on legislation important to the rare disease community. The most powerful tool a legislator […]

RareVoice Awards Gala, Washington DC

On November 14, 2012, RDLA hosted its inaugural RareVoice Awards Gala to honor and thank Members of Congress, Congressional Staff and Patient Advocates for their advocacy to ensure The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187 included provisions that spur the development of lifesaving treatments for rare disease patients.  Abbey Meyers, Founder of the National Organization for […]

National Pediatric Research Network Act (H.R. 6163/S. 3461)

SENS. SHERROD BROWN, ROGER WICKER INTRODUCE BIPARTISAN LEGISLATION to Strengthen and Expand Pediatric Research Despite Children Making Up 20 Percent of the U.S. Population, Just Five Percent of NIH’s Research is Dedicated to Pediatric Research; National Pediatric Research Network Act Expands NIH’s Investments into Pediatric Research, Including Rare Diseases

President Signs Rare Disease Legislation into Law

FDA User Fee Bill Promises Real Hope for Rare Disease Patients
Major Wins for the Grassroots Patient Community
July 9, 2012, Washington, DC —– Rare Disease Legislative Advocates (RDLA) hails the President’s singing of The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, landmark legislation that will encourage the development of new treatments for the 30 million Americans suffering from rare diseases.