Prosensa is a biotech company located in the Netherlands. They are developing and taking to trial the exon skipping work done by Dr. Judith van Deutekom at Leiden University.

Prosensa received the Orphan Drug Designation from the FDA in December 2005. They have also applied for the first ever human trial of exon skipping. This will be an intramuscular trial and will provide proof of principle for future clinical trials.

Exon skipping is a technology which tricks the the dystrophin gene into producing the missing protein which causes Duchenne muscular dystrophy.

PTC is a biotech company located in New Jersey which has pioneered the use of small molecule drugs for various diseases. CureDuchenne was one of the early contributors, through Parent Project, for the high throughput screening of compounds to find oral drugs which will up-regulate or down-regulate other genes in the body to compensate for the lack of dystrophin protein.

We look forward to continuing our funding as PTC moves into the next stage of research which will be drug optimization. This stage will hopefully translate several of these compounds into drugs suitable for animal and ultimately, human testing.

We are monitoring other potential funding projects which include:

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ANTI-INFLAMMATORY AGENTS – Reduce muscle damage

CALPAIN INHIBITORS – Reduce muscle damage

We are watching the work of Dr. Jeff Chamberlain and other top scientists as they continue to make progress toward what could be a long lasting therapy, or even a cure.