4 ms·

I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene tr

by Chriky 9y ago

I have Haemophilia A, I was actually offered a place on an earlier trial although I didn't do it. It was explained to me that you would not need further gene treatments even though it targeted adult cells. I think most cells are made by mitosis and don't come from stem cells. Here's a screenshot of the explanation I received when I was invited on the trial -https://i.imgur.com/H329Vgv.jpg https://i.imgur.com/H329Vgv.jpg > "By using BMN 270 to provide the functional genes, it is expected that you may not require treatment for Haemophilia A, as often or at all." I don't really want to upload the whole Participant Information Sheet because it contains some of the doctor's personal details.

seren 9y ago
Thanks for clarifying my misconceptions !
jessriedel 9y ago
Thanks for sharing! Can I ask what considerations went into your decision? Will you be pursuing this now that there is more evidence that it's effective?
- Chriky 9y ago
  I mean, obviously I regret it now, but at the time I had no idea it would work. I'm sure the next trial will be extremely oversubscribed! I think my trial was actually for a low dose version to test safety of the vector and it didn't have any clinical effects. Its quite incredible though, there's only a few thousand people in the UK with haemophilia - this trial just cured like 0.5% of them
  - toomuchtodo 9y ago
    Would you mind keeping us updated if you pursue the next clinical trial? Happy to kick in Patreon money for your time. (Not a wierdo, just deeply interested in Curing All The Things).
  - igravious 9y ago
    What wonderful news for you. I understand your caution. Now your whole life will be changed. Some people are so brave.
  - jessriedel 9y ago
    Yea, I definitely was not suggesting it was a mistake with the info you had. Most clinical trials don't work. Thanks again for the info. Best of luck!
akuafo 9y ago
My son also has hemophilia A and I'm fascinated by the possibility that he may someday join one of these trials. The increase in the pipeline of gene therapies just in the past few months has been incredible. Here are the ones I'm tracking: BioMarin valoctocogene roxaparvovec (BMN-270) Spark SPK-8011 Sangamo (Pfizer) SB-525 Bioverativ BIVV-001 Shire SHP-654 (BAX-888)
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by Chriky 9y ago

seren 9y ago

Thanks for clarifying my misconceptions !

jessriedel 9y ago

Thanks for sharing! Can I ask what considerations went into your decision? Will you be pursuing this now that there is more evidence that it's effective?

Chriky 9y ago
I mean, obviously I regret it now, but at the time I had no idea it would work. I'm sure the next trial will be extremely oversubscribed! I think my trial was actually for a low dose version to test safety of the vector and it didn't have any clinical effects. Its quite incredible though, there's only a few thousand people in the UK with haemophilia - this trial just cured like 0.5% of them
- toomuchtodo 9y ago
  Would you mind keeping us updated if you pursue the next clinical trial? Happy to kick in Patreon money for your time. (Not a wierdo, just deeply interested in Curing All The Things).
- igravious 9y ago
  What wonderful news for you. I understand your caution. Now your whole life will be changed. Some people are so brave.
- jessriedel 9y ago
  Yea, I definitely was not suggesting it was a mistake with the info you had. Most clinical trials don't work. Thanks again for the info. Best of luck!

akuafo 9y ago

My son also has hemophilia A and I'm fascinated by the possibility that he may someday join one of these trials. The increase in the pipeline of gene therapies just in the past few months has been incredible. Here are the ones I'm tracking: BioMarin valoctocogene roxaparvovec (BMN-270) Spark SPK-8011 Sangamo (Pfizer) SB-525 Bioverativ BIVV-001 Shire SHP-654 (BAX-888)

deleted 9y ago

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