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What We're Funding
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PTC in Phase 2 Trials of
Promising Drug Therapy
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PTC 124
No one can say that Duchenne is still a hopeless disease. PTC is in Phase 2 trials for a drug that holds great promise of halting the progression of Duchenne. PTC124 allows the cellular machinery to read through premature stop codons in mRNA, and thereby enables the translation process to produce full-length, functional proteins. This particular drug will only be effective on approximately 10% of DMD boys who have a stop codon mutation, but it appears that PTC has made a big step forward and offers hope to all Duchenne families.
PTC’s Small Molecule Initiative
PTC has also developed a technology for screening potential drug compounds which could either up-regulate or down-regulate various genes in the body which could compensate for the lack of dystrophin, the missing protein in DMD boys.
CureDuchenne was one of the largest contributors to the first stage of this research and the results were very positive. PTC came up with “hits” on 5 different gene targets. The next step for PTC is to take these compounds and turn them into drugs so they may be tested in a pre-clinical setting. Other companies and institutions are developing similar high-throughput screening techniques, including UCLA. CureDuchenne will monitor the development of this research and will fund the most promising project.
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