Urea Cycle Disorder Discussion Board

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Anyone have a child (or themselves) in the new medication trial that will hopefully replace Buphenyl? My daughter has been asked to participate and just wanted some first hand experiences....
How is the taste - is it easy to take? Had any issues or concerns?
I am leaving the decision to Emma - since it is her body and she is the one who has to go through the extra bloodwork etc - at any rate any experiences you could share would be great!
Thanks!!
RD

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New Medical Trial HPN 100 — Ofishallie, Sun Jul 4 00:59

Hey Gang, I am in the trial. Was first in double blind in February and then started trial in March 1, 2010. Drug is 100% better than the old buphenyl. Does not burn esophagus or stomach. I go in once ... more
- Re: New Medical Trial HPN 100 — nadia, Mon Jul 5 04:18
  
  Hi Ofi how much of the medicine do you have to take a day? is the dosage a lot oless than sodium phenylbutyrate and how many times a day do you have to take it? my son has to take his 3 times a day
- - HPN Dosage — Ofishie, Tue Jul 13 12:10
    
    THe HPN-100 is liquid. I take .580ml with 3 meals a day. Better than 48 pills.
  - - Re: HPN Dosage — nadia, Tue Jul 13 16:26
      
      i am shocked you have to take so much medicine. i suppose thats the down side to these illnesses the medicine alweays increases and never decreases. i spopke to our team here the nurses hav not heard ... more
    - - Drug dosages — Cindy, Wed Jul 14 11:13
        
        Hi Nadia, The dosage of sodium phenylbutyrate increases with weight. The dose is based on mg/kg per day of body weight, so as one increases in weight,the dosage increases. Once the individual gets to ... more
      - Re: Drug dosages — nadia, Wed Jul 14 14:43
        
        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
        
        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... more
  - for nadia — marta, Tue Jul 6 11:15
    
    Hi Nadia, i heard even though i am not on it that it is like one teaspoon once a day. this is what my metabolic dr said anyway. are you part of the trail? I HEARD that it is hard to be a part of.... more
  - - Re: for nadia — nadia, Tue Jul 6 14:03
      
      no we are not part of the trial, we're in england and havnet even heard anything about this trial i am going to ask our metabolic doctor next time we go to clinic. does anyone know how long it will... more
    - - for nadia — marta, Wed Jul 7 11:24
        
        Nadia, i know it goes through it's trai period then it has to be fda approved before insurance will pay for the medicine. not sure though. marta
Clinical Trials for HPN-100 — Cindy, Fri Jul 2 22:00

Hi Rebecca, The adult patients and parents of children in the clinical trial report to me that they are very happy with HPN-100 and cannot imagine going back to Buphenyl. The point of encouraging the ... more
- Adult Trial — Ofishy, Tue Jul 13 12:12
  
  I am on the adult trial. Any one that has questions can reach me at ucdsurvivor@gmail.com You can use this Cindy.
- - Adult Trial — Cindy, Tue Jul 13 12:30
    
    Thanks much! Can you email me off list?
new medication trail — Anonymous, Fri Jul 2 12:09

Hi Rebecca, I am a women born with citrullinemia. i was on buphenyl until it caused me me ulcers and problems with taking it so more times then not i would not take it and end up in the hospital with ... more
Re: New Medication Trial — nadia, Fri Jul 2 06:25

hi wat is the new medicine please? whats the name
- New Med — Rebecca, Fri Jul 2 15:52
  
  The new medicine is in the clinical trial stage - not approved by FDA yet. It is called HPN-100. It is an oil based medication - we are possibly going to enter the study just wondering if anyone had... more
- - Re: New Med — nadia, Fri Jul 2 17:51
    
    thanks for the info thats really good news as it is hard to administer the medicines as we have experienced. my son has been taken off phenylbutyrate and ony had sodium benzoate and arginine. sodium... more

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