PROGRESSIVE PSEUDOHYPERTROPHIC MUSCULAR DYSTROPHY

Report of Results of Treatment with Adrenalin and Pilocarpin with an Analysis of Twenty-eight Cases

¹ The Shriners' Hospital for Crippled Children, Springfield

From the data presented we feel justified in reaching the following definite conclusions.

1. Progressive pseudohypertrophic muscular dystrophy is a more common syndrome than is commonly appreciated, having an incidence of approximately six per 100,000 total population in the New England states.

2. The disease is wide-spread, occurring throughout the civilized world.

3. The recently acquired knowledge of the double enervation of skeletal muscles has opened up a new conception of the pathology and pathogenesis of the disease.

4. Contrary to the original conception that the disease is primarily of the muscle substance, there is evidence to believe that there is a primary lesion of the mid-brain, affecting the autonomic nervous system.

5. The endocrine disturbances frequently reported are not an essential part of the disease, but are due to derangement of the autonomic enervation of these glands.

6. The endocrine dysfunctions are seen most commonly in the less typical cases.

7. The complicating central nervous system disorders are due to a more extensive involvement in the primary lesion.

8. In some cases there is suggestive evidence that the primary lesion may be caused by an acute infection. In the majority of cases, however, there is no direct evidence of this.

9. The natural course of the disease is fairly constant,—with onset of symptoms appearing between the ages of two and four, and progressing to inability to walk at between eight and twelve. In four to six years more the use of the arms is lost, and death from an intercurrent infection occurs after another two to three years, at an age of from fifteen to twenty.

10. Previous therapeutic measures have been almost uniformly without effect.

11. Hyperdermic injections of adrenalin and pilocarpin have been of symptomatic temporary benefit in every case.

12. In the extremely advanced cases this improvement has been of more scientific interest than of practical value.

13. In hopelessly bed-ridden cases definite increase of independence by the improved use of the arms has been obtained.

14. In a comparatively early case a symptomatic and functional recovery has been obtained.

15. No claim can be made for the permanency of the improvement. It is our purpose to keep the cases under observation, and report subsequently on their ultimate outcome.

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