When I was misdiagnosed and placed in the Limb Girdle muscular dystrophy category, I was hoping that the gene therapy the researchers were trying at the time would be successfull. This article just released shows how far gene therapy has come and I think there is great hope for people with HIBM. I have attached the link of the complete article.
"The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies".
http://www.labspaces.net/121546/New_gene_transfer_strategy_shows_promise_for_limb_girdle_and_other_muscular_dystrophies
This is a forum for GNE Myopathy where I discuss the challenges and insights I encounter on my journey in living with a progressively "weakening" disease. GNE Myopathy is also known as Hereditary Inclusion Body Myopathy (HIBM). I will invite others to share their stories, tips, and comments. I will discuss potential treatments, clinical trials, current research, and resources available for patients with GNE Myopathy.
tara
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1 comment:
Hi Tara,
How do I become a follower of your blog?
Kristine
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