Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and, in some cases, provide a functional cure. However,…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can…
Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these…
Epi Data Slicer
Epi Data Slicer
Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can…
With the increasing number of approved innovative, high-cost gene therapies, value assessment and novel approaches to reimbursement are rapidly gaining importance. To assist developers of gene…
As the number of gene therapy approvals rises, novel approaches to value assessment and reimbursement schemes for these innovative, high-cost treatments are rapidly gaining importance. Developers…
DRG Epidemiology’s coverage of RP comprises epidemiological estimates of key patient populations across the major mature pharmaceutical markets (the United States, France, Germany, Italy, Spain…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. There is a high unmet need for…