Can a Genetic Disease Be Cured?
A Mother's Dilemma
Department of Biological Sciences
University at Buffalo
In this discussion case, parents must decide whether or not to enroll their sons in an experimental treatment program designed to alleviate the symptoms of muscular dystrophy. The case explores the genetics and physiology of the disease as well as the issues involved with pharmaceutical research. The case has been used in both majors’ and non-majors’ biology courses, where it is introduced during a unit on the cardiovascular and muscular systems.
- To learn about normal muscle and nerve function.
- To understand the genetic basis underlying muscular dystrophy.
- To understand the pathology of muscular dystrophy.
- To learn about the guidelines and reasons behind FDA regulations for introducing new pharmaceutical agents.
- To understand the role (obligatory, in many cases) of animal models in disease and pharmaceutical research.
- To appreciate the role that money and grants play in scientific advances.
- To gain insight into the emotional problems involved in family health care decisions.
KeywordsMuscular dystrophy; neurodegenerative disease; genetic disease; pharmaceutical research; animal model; drug development; drug approval process; muscle; Food and Drug Administration; FDA; health care decisions; bioethics; sociology of science
Topical AreasEthics, Scientific method, Regulatory issues, Social issues
Educational LevelHigh school, Undergraduate lower division, Undergraduate upper division
Type / MethodsDilemma/Decision, Discussion
Subject HeadingsBiology (General) | Medicine (General) | Physiology | Anatomy | Genetics / Heredity |