Meet, discuss & support other patients or families living with Cystinosis. Participate in group discussions. Contribute to topics, or just share what's on your mind
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robpleticha | Cystagon | 14 days ago | Originally written in English Delayed release (DR) cysteamine (RP103)...
The results of the Phase III trial seem to be positive (bit.ly/ojOgaG), what does this mean to patients? When can patients realistically expect this new drug to be approved by the drug regulatory agencies?
robpleticha | 16 days ago | Originally written in English Sky Dive & School Fundraising Events Top £3,000
I am amazed at how Cystinosis patients, families, and their entire communities come together to help raise money for research.
Here is another great example of that via Cystinosis UK:
he National Institutes of Health have Genetic and Rare Disease (GARD) Information specialists that may be able to help you find information.
The GARD Information Center does not provide genetic counseling and does not offer diagnostic testing, referrals, medical treatment or advice.
There is a phone number and online form found here:
Information in Spanish and English. You don’t have to be living in the US to ask a question.