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When New Treatments Come with Big Hopes and a Big Price Tag

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Snapshot Author(s)

Name and Degree
Personal Genetics Education Project (

Spinal Muscular Atrophy is a rare genetic disorder, characterized by a loss of motor function. Researchers have developed treatments for people with this rare genetic disorder, and this lesson highlights families and patients who are excited about new treatments but also discusses how the cost of these new advances prohibit access for people who might benefit.

rare genetic disorder
Cost of Illness
Spinal Muscular Atrophy