Genetic Disorder Cluster Muscular Dystrophy Drug Development Report 2 Genetic Disorder Cluster Muscular Dystrophy Drug D | Page 2

The size of these two pipelines ranges from just six in BMD to 108 in DMD. The majority
of pipeline products for DMD target dystrophin which, as mentioned, is the causative
gene for the disease .
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Scope
- Which companies are the most active within the pipeline for genetic
disorder therapeutics?
- Which pharmaceutical approaches are the most prominent at each stage
of the pipeline and within each indication?
- To what extent do universities and institutions play a role within this
pipeline, compared to pharmaceutical companies?
- What are the most important R&D milestones and data publications to
have happened in the field of genetic disorder therapeutics?
Sample Request : Genetic Disorder Cluster Muscular
Reasons to buy
- Understand the overall pipeline, with an at-a-glance overview of all
products in therapeutic development for each indication
- Assess the products in development in granular detail, with an up-to-date
overview of each individual pipeline program in each indication, and a
comprehensive picture of recent updates and milestones for each.