Navigation Links
Parent Project Muscular Dystrophy Endorses Senate Version of Prescription Drug User Fee Act After Patient Voice Strengthened

HACKENSACK, N.J., May 24, 2012 /PRNewswire-USNewswire/ -- As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, Parent Project Muscular Dystrophy (PPMD) is pleased to offer its enthusiastic endorsement of the legislation.


Over the past several months, PPMD – the leading advocacy organization fighting to end Duchenne muscular dystrophy (Duchenne) – has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the Prescription Drug User Fee Act or PDUFA. A number of similar provisions are also included in a House bill expected to be voted on by that chamber next week.

"The provisions included in the Senate user fee bill will help expand the patient voice when key decisions are being made, an issue of grave importance to our community and to the larger rare disease community," PPMD President Pat Furlong said.  "The provisions will also provide the FDA with additional tools to more quickly review potential therapies for life-threatening conditions, including Duchenne."

The organization has also supported expanded external expert engagement on key issues such as benefit/risk and trial design, and has urged the FDA to accelerate the timing of various rare disease-focused activities.

"As Congress works to finalize the user fee legislation, PPMD calls upon the U.S. House of Representatives to amend their version of the user fee bill to mirror the patient representative provisions contained in the Senate legislation," Furlong said.

The legislative provisions that strengthen the patient voice throughout the FDA review process parallel the FDA Policy position statement adopted by the PPMD Board of Directors in February, and align with the Congressional request made by the PPMD community during the 2012 Advocacy Conference in Washington, DC.

About Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy.  Our mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community. PPMD is headquartered in Middletown, Ohio with offices in Hackensack, New Jersey.  For more information, visit

SOURCE Parent Project Muscular Dystrophy
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. A Sociolinguistic Analysis of In-Office Dialogue Reveals Psychiatrists and Parents Have Significantly Different Perceptions on ADHD and Psychiatric Comorbidities
2. An Open Letter to Parents, Students, School Administrators, & Health Care Officials from Soapopular(R)
3. Survey Shows Parents Best Intentions Out of Step with Expert Recommendations in Protecting Kids from Flu
4. Parents Make Donations to National Autism Association for Cutting-Edge Research
5. Unprecedented 8th Year Start Up of National Direct AmeriCorps Program Access to Care Through Service (ACTS) Parent, Lead Organization The Philadelphia AIDS Consortium and World Health Care Infrastructures
6. Photos: NIH Launches New Web Site for Parents on Medical Research Studies for Children
7. The COSHAR Foundation Urges Parents to Ensure Children Are Up-to-Date on Vaccinations
8. Transparent Inc. to Introduce Cell-able, a Unique Human Hepatocyte Array, to U.S. Market at ISSX Meeting
9. National Foundation for Infectious Diseases (NFID) Survey: Parents More Flu Aware and More Likely to Get Kids Vaccinated
10. IQAir HealthPro Plus Selected as a Top 10 Product for Parents
11. deCODE Discovers a Major Risk Factor for Type 2 Diabetes Dependent on Parent of Origin
Post Your Comments:
(Date:6/23/2016)... Research and Markets has announced the ... 2016 - Forecast to 2022" report to their offering. ... up to date financial data derived from varied research sources ... with potential impact on the market during the next five ... comprises of sub markets, regional and country level analysis. The ...
(Date:6/23/2016)... -- Bracket , a leading clinical trial technology and ... platform, Bracket eCOA (SM) 6.0, at the 52 nd ... 2016 in Philadelphia , Pennsylvania.  A demonstration ... of its kind to fully integrate with RTSM, will be ... is a flexible platform for electronic clinical outcomes assessments that ...
(Date:6/23/2016)... today announced the establishment of Astellas Farma Colombia (AFC), a new affiliate with operations headquartered in Bogota. ... ... ... ... Astellas is a ...
Breaking Medicine Technology:
(Date:6/25/2016)... , ... June 25, 2016 , ... Conventional wisdom preaches ... success. In terms of the latter, setting the bar too high can result in ... than just slow progress toward their goal. , Research from ...
(Date:6/24/2016)... ... , ... Those who have experienced traumatic events may suffer from a complex ... as drug or alcohol abuse, as a coping mechanism. To avoid this pain and ... a traumatic event. , Trauma sufferers tend to feel a range of emotions, from ...
(Date:6/24/2016)... ... ... was in a crisis. Her son James, eight, was out of control. Prone to extreme ... “When something upset him, he couldn’t control his emotions,” remembers Marcy. “If there was ... other children and say he was going to kill them. If we were driving ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... Global law ... magazine’s 2016 Legal Elite. The attorneys chosen by their peers for this recognition are ... , Seven Greenberg Traurig Shareholders received special honors as members of this year’s Legal ...
(Date:6/24/2016)... (PRWEB) , ... June 24, 2016 , ... Comfort Keepers® ... American Cancer Society and the Road To Recovery® program to drive cancer patients to ... and other adults to ensure the highest quality of life and ongoing independence. ...
Breaking Medicine News(10 mins):