Ryan Fischer's Blog – July 2012 Archive (3)


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

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