Scope of report The report provides a snapshot of the global therapeutic landscape of Duchenne Muscular Dystrophy. The report assesses Duchenne Muscular Dystrophy pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), and molecule type. The report reviews Duchenne Muscular Dystrophy pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources. The report covers pipeline products based on various stages of
Scope of report The report provides a snapshot of the global therapeutic landscape of Cystic Fibrosis. The report assesses Cystic Fibrosis pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), and molecule type. The report reviews Cystic Fibrosis pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources. The report covers pipeline products based on various stages of development for Cystic
Scope of report The report provides a snapshot of the global therapeutic landscape of Amyotrophic lateral sclerosis. The report assesses Amyotrophic lateral sclerosis pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), and molecule type. The report reviews Amyotrophic lateral sclerosis pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources. The report covers pipeline products based on various stages of
Scope of report The report provides a snapshot of the global therapeutic landscape of Alpha 1-antitrypsin deficiency. The report assesses Alpha 1-antitrypsin deficiency pipeline based on highest phase of development, type of sponsor, mechanism of action (MoA), route of administration (RoA), and molecule type. The report reviews Alpha 1-antitrypsin deficiency pipeline by companies and universities/research institutes based on information derived from company and industry-specific sources. The report covers pipeline products based on various stages of
Scope of report The report provides brief overview of Rett syndrome. The report highlights major unmet medical need in Rett syndrome diagnosis, patho-physiology, treatment & in prognosis. This report highlights FDA guidance for drug development in orphan disease & applicability of this guidance for Rett syndrome in terms of sample size, trial design & outcome measures (clinical endpoints). The report provides epidemiology data of Rett syndrome in US, Europe & Japan. The report maps pipeline
