The most important unmet need is the lack of therapeutic options, because for a few neuromuscular disorders we do have a very good therapy, but for the majority of neuromuscular conditions we are still lacking very good molecules or gene therapy, for instance. So the most important medical unmet need is definitely the lack of therapy, right? And then another important unmet need in neuromuscular disease patients is that the awareness, the knowledge of this kind of disorder, being rare, is not so obvious, and so many times these patients have to go through all these steps to get the diagnosis because they jump from one office to another until they find an expert on the rare neuromuscular disease...
The most important unmet need is the lack of therapeutic options, because for a few neuromuscular disorders we do have a very good therapy, but for the majority of neuromuscular conditions we are still lacking very good molecules or gene therapy, for instance. So the most important medical unmet need is definitely the lack of therapy, right? And then another important unmet need in neuromuscular disease patients is that the awareness, the knowledge of this kind of disorder, being rare, is not so obvious, and so many times these patients have to go through all these steps to get the diagnosis because they jump from one office to another until they find an expert on the rare neuromuscular disease. So it’s a long journey most of the time to get the correct diagnosis. So we need to implement awareness and knowledge in this chapter of medicine. The field of gene therapy in neuromuscular disorders is growing as well. We already have some gene therapy approved for selected neuromuscular disorders. You know, the most famous one is SMA, where we can really change the natural history of the disease now, but there are a lot of phase one and phase two clinical trials already ongoing for a lot of neuromuscular diseases, and I’m pretty sure that within five to ten years we will have additional gene therapies for additional neuromuscular disorders.
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