[HTML][HTML] Treatment of facioscapulohumeral muscular dystrophy with Denosumab

SS Lefkowitz, DL Lefkowitz, J Kethley - The American Journal of …, 2012 - ncbi.nlm.nih.gov
SS Lefkowitz, DL Lefkowitz, J Kethley
The American Journal of Case Reports, 2012ncbi.nlm.nih.gov
Background: Facioscapulohumeral muscular dystrophy (FSHD) is the 3 rd most common
form of muscular dystrophy. Effective treatments for any of the muscular dystrophies have yet
to be realized. This report describes such a treatment. Case Report: A 66 year old female
was diagnosed with osteoporosis. She had been diagnosed with FSHD muscular dystrophy
a number of years previously by both genetic and clinical studies. Following a 2 year course
with Forteo for osteoporosis, she was given an injection of Denosumab (Prolia) to maintain …
Summary
Background:
Facioscapulohumeral muscular dystrophy (FSHD) is the 3 rd most common form of muscular dystrophy. Effective treatments for any of the muscular dystrophies have yet to be realized. This report describes such a treatment.
Case Report:
A 66 year old female was diagnosed with osteoporosis. She had been diagnosed with FSHD muscular dystrophy a number of years previously by both genetic and clinical studies. Following a 2 year course with Forteo for osteoporosis, she was given an injection of Denosumab (Prolia) to maintain her bone density. By 24 hours, she exhibited increased strength and a dramatic reduction of her dystrophic symptoms eg she could walk unassisted in high heels. She was able to accomplish other things that had not been possible for a number of years. After approximately 5 weeks she gradually lost the newfound strength with a complete loss by about 6 weeks. A second injection of Denosumab resulted in the same effect, ie reversal of symptoms and increased functionality. A number of measurements and videos were taken to establish the beneficial effects of Prolia for future studies. This was repeated with a 3 rd and 4 th injection in order to establish the unequivocal beneficial effects on muscular dystrophy.
Conclusions:
Further studies will be required to establish Denosumab as a major “front line” treatment for this disease and possibly other muscular dystrophies.
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