Ryan Fischer's Blog (94)

Implementing PDUFA

With Congress decamping from Washington and not expected to return until after the November election, we thought we'd revisit one of the few bright spots of bipartisanship this Congress – the enactment of the latest FDA user fee law (PDUFA), known as the FDA Safety and Innovation Act or FDASIA. (Washington loves a good acronym).



Added by Ryan Fischer on September 26, 2012 at 10:00am — No Comments

Senate and House Continue to Send Message Against CDC Consolidation

Thank you to everyone who reached out to their Senate and House members about stopping consolidation of vital Duchenne programs at the CDC! Members of both the…


Added by Ryan Fischer on August 9, 2012 at 10:09pm — 2 Comments

PPMD Endorses The National Pediatric Research Network Act

(photo credit)

In a letter to Senators Brown and Wicker (PDF), PPMD officially endorsed the National Pediatric…


Added by Ryan Fischer on August 9, 2012 at 9:46pm — No Comments


A team of UCLA scientists, clinicians and clinical trialists led by Drs. Stan Nelson and Carrie Miceli have teamed with AVI and Dr. Craig McDonald to bring a novel combination therapy forward for use in DMD and sent a proposal to the California Institute of Regenerative Medicine (CIRM). It involves the UCLA discovery of an already FDA approved drug to boost exon skipping activity when used in combination with anti-sense oligo-nucleotides for Duchenne. Duchenne patient skin cells…


Added by Ryan Fischer on July 23, 2012 at 5:00pm — 2 Comments

Landmark FDA legislation becomes law

Major win for the Duchenne community

PPMD applauds the President for signing into law the Prescription Drug User Fee Act also known as The Food and Drug Administration Safety and Innovation Act. Most recently, PPMD worked with the Everylife Foundation for Rare Diseases and Genetic Alliance to organize a letter to…


Added by Ryan Fischer on July 9, 2012 at 6:00pm — No Comments

Action Alert! Contact your Senators to help save vital Duchenne programs at the CDC

We need your voice to help save critical Duchenne related programs at the Center for Disease Control (CDC)!…


Added by Ryan Fischer on July 9, 2012 at 12:00pm — No Comments

Results from 2012 Advocacy Efforts- Success on Senate Appropriations!

Last week, the Senate Appropriations Committee approved its version of the bill that would fund many key agencies, including the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC), in FY 2013.  Thanks to the hard work of PPMD advocates, the bill reflects a number of our priorities.


At the CDC, Senators strongly objected to the administration’s proposal to consolidate the many programs within the National Center on Birth Defects…


Added by Ryan Fischer on June 22, 2012 at 1:14pm — 1 Comment

PPMD Leads Critical Letter for Rare Disease Community

Parent Project Muscular Dystrophy (PPMD) has teamed up with EveryLife Foundation for Rare Diseases and Genetic Alliance to galvanize the rare disease community and send a strong message to…


Added by Ryan Fischer on June 13, 2012 at 3:00pm — No Comments

PDUFA Sails Through House of Representatives

Following recent overwhelming passage in the Senate, The Prescription Drug User Fee Act comes closer to becoming law after swift passage in the House of their version of the bill last night by a vote of 387-5. Both bills now move to conference for reconciliation.…


Added by Ryan Fischer on May 31, 2012 at 10:30pm — No Comments

PDUFA Moves Closer to Passage

Few pieces of legislation may be as peculiar – and as important to the development of drugs and devices in this country – as the package of bills known as Food and Drug Administration (FDA) Prescription Drug User Fees Acts, known as PDUFA, or user fees for short. Born out of a frustration – amplified by the lack of therapies to respond to the AIDS epidemic – that FDA was taking too long to review drug applications, Congress enacted the first such law 20 years ago, in 1992. The law set out…


Added by Ryan Fischer on May 17, 2012 at 10:28am — No Comments

Tis the Season, for FDA legislation called PDUFA

The Senate and House are moving closer to taking action on critical legislation focused on the Food and Drug Administration called PDUFA. Every five years Congress must pass a package of policies, known as user fees, that dictate the terms for the pharmaceutical and biotechnology and related industries to help fund the FDA and its review of candidate therapies. The bills…


Added by Ryan Fischer on April 24, 2012 at 10:30am — No Comments

Take Action: Ask Your Representative to Co-Sponsor FAST Act

We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications.…


Added by Ryan Fischer on March 30, 2012 at 9:30am — No Comments

Advocacy: Get Active!

With deficit reduction remaining a top priority in Congress and a $1.2 trillion spending reduction on the horizon, 2012 will be a critical year to protect Duchenne research and public health priorities. For this to happen, we need your voice on Capitol Hill now more than ever before. Whether that be by joining us in Washington at the Annual Advocacy Conference or advocating from home by contacting…


Added by Ryan Fischer on January 5, 2012 at 2:00pm — No Comments

So Much Happening this Weekend....

Greetings Friends!


We have a very busy weekend ahead of us, with some fantastic events happening in the community!

I wanted to take a moment to highlight these fun and very different fundraisers taking place over the weekend.

Big shout out to all those who are organizing events (not just these events- all…


Added by Ryan Fischer on October 21, 2011 at 1:30pm — No Comments

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