This is an important question and I would like to send this out to our broader community to ensure that we can get an answer for you. Stay tuned - please also join us at w ww...
I usually access the Orphanet database that can be found within www.crdnetwork.org/resources. We link directly to this database - they have updated information on different drugs and therapies in dev elopment...
Rare Disease is defined as having an affected audience of 200,000 or less. There are 7,000 identified rare diseases with about 5600 defined as Ultra-Rare, affecting less then 6,000 people. There are a...
There are two identified rare disorders that may apply to your daughters condition. We usually start searching within the Orphanet database which is one that we believe to be the most comprehensive - please...
I am not sure, but I would recommend you contact Ms. Christine Brown, Executive Director of National PKU Alliance they will be able to answer most of your questions specific to PKU. www.npkua.org christine...