Primary α-sarcoglycan deficiency responsive to immunosuppression over three years

A. M. Connolly, A. Pestronk, S. Mehta, M. Al-Lozi

Research output: Contribution to journalArticlepeer-review

41 Scopus citations


An 8-year-old girl developed weakness over 2 years and an elevated creatine kinase. The biopsy was most consistent with an active dystrophy with many inflammatory cells present. A trial of immunosuppression was started. In the first 2 months of treatment with prednisone, she had functionally and quantitatively significant improvement in her proximal strength. Over 3 years of treatment she maintained stable strength. Subsequent genetic studies showed that she had primary α-sarcoglycan deficiency. The timing and the degree of benefit in strength were similar to that seen in boys with Duchenne muscular dystrophy who are treated with prednisone.

Original languageEnglish
Pages (from-to)1549-1553
Number of pages5
JournalMuscle and Nerve
Issue number11
StatePublished - 1998


  • Adhalin
  • Duchenne muscular dystrophy
  • Limb-girdle muscular dystrophy type 2D
  • Polymyositis
  • α-sarcoglycan


Dive into the research topics of 'Primary α-sarcoglycan deficiency responsive to immunosuppression over three years'. Together they form a unique fingerprint.

Cite this