The information that treatment after the onset of the disease may be beneficial, comes on the heels of new data on what is possibly the best candidate for SMA cure so far.
The drug I'm writing about is ISIS SMNrx, which is supposed to boost the production of the "backup" SMN gene. While the majority of the SMN protein is produced by the SMN1 gene, there is also the SMN2 gene that normally only produces a fraction of the SMN protein needed. ISIS SMNrx might stimulate the SMN2 gene enough to provide us with a normal dose of the protein we lack. The drug is currently still in preclinical stages, but I'm keeping my fingers crossed.
Detailed information.
The drug I'm writing about is ISIS SMNrx, which is supposed to boost the production of the "backup" SMN gene. While the majority of the SMN protein is produced by the SMN1 gene, there is also the SMN2 gene that normally only produces a fraction of the SMN protein needed. ISIS SMNrx might stimulate the SMN2 gene enough to provide us with a normal dose of the protein we lack. The drug is currently still in preclinical stages, but I'm keeping my fingers crossed.
Detailed information.
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