Replies to post #69478 on Biotech Values

[DewDiligence]

Welch has always struck me as a straight shooter. Was he crossed up by Roche, or did he simply misinterpret the question about the INFORM-1 design on the recent webcast?

[genisi]

>I thought BMT was not an option for MPS VI. A little more research and I found it is an option in some, do you know how common?<

In general, bone marrow transplant (BMT) has been of particular interest in treating patients with MPS who are at risk for neurologic disease. Since there are no neurological manifestations of the condition, the majority of MPS VI patients are not candidates for BMT and it is used in a small minority of patients. Naglazyme is a better option.

>Am I right in thinking BMT is not an option in Morquio too. <

Yes, BMT was tried but with disappointing results and is not recommended as a treatment for Morquio disease.