Track topics on Twitter Track topics that are important to you
Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017
This report provides an overview of the genetic disorders pipeline landscape, specifically focusing on muscular dystrophies. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for Becker and Duchenne Muscular Dystrophy BMD and DMD and features dormant and discontinued projects. Both indications covered are inherited muscular dystrophies, a group of genetic, degenerative diseases primarily affecting voluntary muscles.
BMD, the first type of muscular dystrophy, leads to symptoms include pain and sensation, difficulty with running, hopping, and jumping, toe walking, breathing problems, cognitive problems and frequent falls. DMD is a condition which causes muscle weakness, and is an Xlinked disorder. The gene for DMD is present on the X chromosome. It codes for a protein named dystrophin. Dystrophin is essential for providing structural support inside muscle cells. Symptoms usually appear before age 6 and may appear as early as infancy. They include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking.
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. However, a number of products act on other molecular targets such as growth differentiation factor 8, nuclear factor kappa B and utrophin. The first two of these make up the pipeline for BMD, along with forkhead box protein 1B.
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 RD milestones and data publications to have happened in the field of genetic disorder therapeutics?
Reasons to buy
Understand the overall pipeline, with an ataglance overview of all products in therapeutic development for each indication
Assess the products in development in granular detail, with an uptodate overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each
Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these
Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
Muscular dystrophy is a group of degenerative inherited disorders causing muscle weakness and loss of muscle tissue. The different types are Becker muscular dystrophy, Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy, Facioscapulohumeral mu...
Pharmacy is the science and technique of preparing as well as dispensing drugs and medicines. It is a health profession that links health sciences with chemical sciences and aims to ensure the safe and effective use of pharmaceutical drugs. The scope of...
An anesthesiologist (US English) or anaesthetist (British English) is a physician trained in anesthesia and perioperative medicine. Anesthesiologists are physicians who provide medical care to patients in a wide variety of (usually acute) situations. ...