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XLMTM
This page shows the latest XLMTM news and features for those working in and with pharma, biotech and healthcare.
Audentes’ rare disease gene therapy programme delayed after third patient death
AT132 is an aden-associated virus (AAV) based gene therapy that is designed to deliver the MTM1 gene deficient in XLMTM.
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Astellas makes gene therapy a core focus with $3bn Audentes buy
XLMTM is a rare, genetic neuromuscular disorder that causes muscle weakness that ranges in severity from mild to life-threatening. ... XLMTM only affects approximately one in 40, 000 to 50, 0000 newborn males, so it is a small indication, but gene
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Accession
Accession was a born from a passion and a vision. A passion to harness the power of market access to...
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