Denufosol… the next miracle drug?

Let’s hope so!

There’s been a lot of buzz about the promise of this exciting drug to enter the realm of cystic fibrosis treatments. As it enters its second Phase III trial, the excitement is palpable. Will this change the future of the cystic fibrosis in our youngest population, potentially staving off the effects of lung damage by helping to prevent it in the first place? While the future of denufosol remains unclear for the moment, the long term implications of potentially finding such a drug is giving hope to thousands.  Another reference here.

This news burst follows closely on the heels of another major advancement for those with cystic fibrosis who have what is known as nonsense mutation (approximately 10% of the CF population).  A drug known as PTC124 continues to show promise for these patients as well as some patients with a certain form of Muscular Dystrophy.

In hopes of continuing the momentum and helping to find a cure, I have once again activated my team, Ally’s Allies,  to join the Cystic Fibrosis Foundation’s Great Strides fundraiser.  Join my team, form a team of your own, or simply donate.  Your support makes the difference for these and other drugs to be discovered and used to help save lives… thank you for your help!