Hi Nadia: The average dosage for the new drug is about 2 teaspoons a day. It is a tasteless, odorless liquid. One of the needs for its development was to address the problem of our children developing feeding aversions because of the horridness of phenylbutyrate (Buphenyl) that many families were facing. It will definitely be a great improvement in quality of life and certainly we will keep pushing for more. We have almost won a huge battle as families to get this drug developed -- almost at the finish line, but it's just one of the battles we face. So, onward we march...thank you to everyone who has supported our efforts to accelerate research and improve our children's and affected adults' lives.
Yes, quite a few families have been through PGD to avoid having an affected pregnancy, although it is not without controversy. Warmly, Cindy (NUCDF)
Hi Cindy, thankyou for your message it will be brilliant once it has been approved and we have it in the UK as we struggled a lot when my son had to take phenylbutyrate. Willt he dosage of the new... more
Buphenyl and New Formulation Cindy,Wed Jul 14 15:18