Myozyme Gets Broad Approval by the FDA (Yahoo!)

April 28, 2006 is a very special day for the Pompe Community. The FDA approved a biologics license application for Myozyme. Bringing to reality that a treatment for Pompe disease is now available for patients.

This is truly a day worth remembering. It’s a big thing for Pompe patients like me. It’s a fulfillment of a life long dream to see a treatment for this dreaded disease. It now gives patients something to look forward to.

While the approval of the FDA is great news, this is just the beginning. We’ll have to see how soon and if all patients will have access to Myozyme. It’s going to be expensive. I just hope that Genzyme, government agencies, patient organizations, other non-profit organizations and patients themselves all work together to make sure that no one suffering from Pompe disease doesn’t have access to Myozyme just because he/she can’t afford it. One of the greatest injustices in the world is people not having access to medicine just because they don’t have money. The treatment is there and the stumbling block is money. It shouldn’t be that way…

Enough of that, it’s a great day!!! Congratulations to Genzyme for getting the product to market. Many thanks to the FDA for approving Myozyme for the broad approval. Thanks to the patient organizations such as “AMDA”: for the tireless effort in pushing for a treatment. Lastly, good luck to all Pompe patients and their families, this is the start of a new life for all of us. Let’s hope it goes well…

Genzyme’s press release can be found “here”: and the FDA’s press release can be found “here”:

Now as Kool & The Gang sang…

bq. Celebrate good times, come on! (Let’s celebrate)
Celebrate good times, come on!
It’s a celebration!
Celebrate good times, come on! (Let’s celebrate)

4 Replies to “Myozyme Gets Broad Approval by the FDA (Yahoo!)”

  1. very interesting. I wish I could see the clinical trials, though.

    dont freak out, i was just doing research when i this site popped up as one of the results. decided to let myself in. *hehe, obviously i was doing research on pompe

  2. I to am a Pompe patient, I’m 39 and have known what it was that I had for about 2 years. It is truely a good day for people effected with this ailment.I hope the treatments live up to my exspectations although I don’t really know anything about it. Take care of yourself.

  3. Hey Laterus, thanks for visiting my site. No, I’m not freaking out.. 🙂 Why are you researching on Pompe?

    Hey Randy, Thanks for dropping by. It is a good day. Well as I always say “Hope for the best and expect the worst”. If it’s any consolation though, from experience, a patient is better with the treatment than without.

  4. just the disease itself. im a med student – just brushing up on some terms i can’t seem to recall.

    err… thanks for NOT freaking out then. haha

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