How are gene therapies expected to revolutionise the treatment of Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is a devastating and fatal neuromuscular genetic disease with no known cure and few treatment options. Since 2014, two antisense oligonucleotide therapies [Exondys 51 (Sarepta Therapeutics) and Translarna (PTC Therapeutics)] have been approved but how are they faring on the market? Find out how key opinion leaders (KOLs) perceive these therapies amongst other conventional treatment approaches. The pipeline for DMD encompasses many novel therapies with diverse mechanisms of action. Find out how the KOLs weigh up key therapy classes including novel exon-skipping compounds, adeno-associated virus (AAV) vector based microdystrophin/minidystrophin gene therapies, myostatin inhibitors, cell therapies amongst others. Which therapeutic strategy do the KOLs think will offer the most promise for this life-threatening condition and how will they impact future treatment pathways?
Learn how KOLs see the market evolving, and how developers could differentiate their marketed and pipeline therapies in KOL Insight: Duchenne Muscular Dystrophy [2018]. Ten of the world's foremost KOLs from the US and Europe provide their candid insights on three marketed products and 16 late- and early-stage pipeline programmes.
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"The way the DMD field is moving is in terms of precision medicine and gene-based approaches. I think gene-based therapy targets are going to really play a big and a critical role."
US Key Opinion Leader
"It's likely that we will be moving from the Duchenne we know now to a modified and hopefully, a significantly milder phenotype but not a complete cure. I would put gene therapy as the one at the top of the list at least in the short-medium term."
US Key Opinion Leader
Marketed Therapies
Pipeline Therapies
KOLs from North America
KOLs from Europe
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