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…
Spinal Muscular Atrophy
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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…
Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive muscle weakness; it is a leading genetic cause of death in infants. SMA is divided into four clinical subtypes…
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…
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…
Muscular dystrophies (MDs) are a spectrum of genetic disorders characterized by muscle weakness that, in severe disease forms, can lead to loss of ambulation and early death. Duchenne muscular…