In recent years, tremendous progress has been made in the diagnosis and treatment of spinal muscular atrophy. This consists of a newborn screening program and a drug program that includes three therapies, including gene therapy. Thanks to the possibility of implementing treatment as early as the pre-symptomatic stage, young patients gain a chance for a fully normal life.
Spinal muscular atrophy is a rare, genetically determined disease. Thanks to bold reimbursement decisions, the model of care for SMA patients is one that people struggling with other diseases would like to see in their therapies.
The drug program became operational in 2019, and all three therapies registered for the treatment of SMA have been reimbursed under it since last September. In addition, a nationwide newborn screening program for spinal muscular atrophy has been in effect since last April. So ...
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