PPMD Community

need to track an old news article from Children's Hospital Boston

Hi,
There is an old news article (2002) suggesting the one kid in Children's Hospital Boston had mild form of DMD. There is a suggestion that a bone marrow treatment given earlier to this kid had actually helped him in his DMD. (http://webweekly.hms.harvard.edu/archive/pressreleases/ch/0802_bone...)

If someone is seeing a Doctor as this hospital can we get some updates on this kid. How is this kid doing now.

If indeed the progressions is much slower in this case then I am connecting the dots and relating this to Ryan's Mesenchymal treatment now.

Fingers crossed.
Tulika

Favorite

0 members favorited this

▶ Reply to This

Replies to This Discussion

Permalink Reply by Eileen DeLong on November 24, 2009 at 3:11pm

Hi,
I found this at http://www.duchenne-information.eu/english.htm:
A stem cell experiment of nature: At the age of one year, a Duchenne boy had received a bone marrow transplantation from his father because of another disease. As he could still walk at 14 years in spite of the reading-frame-shifting deletion of exon 45, it was assumed that stem cells from the bone marrow had provided the information for new dystrophin. Investigation of new bioptic material, however, have now shown that the milder symptoms of the disease are due only to a small extent to the transplant, but mainly to a spontaneous additional deletion of exon 44, so that, in the mRNA, exon 46 follows directly after exon 43. This exon skipping normalizes the reading frame. Dystrophin can again be produced but is shorter and therefore causes the symptoms of a Becker dystrophy (see paragraph on exon skipping). Apparently, after 13 years, a bone marrow transplantation can contribute to some extent to a Duchenne therapy, however not sufficiently so to significantly change the disease (Kunkel, Boston).