Hormone Therapy Enhances Muscle Strength and Function in FSHD Patients

Recent research demonstrates that a combined treatment of growth hormone and testosterone is both safe and well tolerated by men diagnosed with facioscapulohumeral muscular dystrophy (FSHD). The study revealed significant improvements in muscle mass, strength, and mobility following the therapy. This groundbreaking approach offers hope for not only slowing the progression of FSHD but also restoring some lost functions.

Led by Dr. Chad Heatwole from the University of Rochester Medical Center, the clinical trial involved 20 adult men with FSHD who retained the ability to walk independently. Over six months, participants self-administered daily injections of recombinant human growth hormone (rhGH), which promotes cellular growth and regeneration, alongside biweekly testosterone injections aimed at enhancing muscle development.

Throughout the trial, safety was closely monitored through blood tests, and participants’ body composition, walking capacity, and strength were evaluated regularly. After the treatment ended, the participants abstained from hormones for three months, allowing researchers to assess the longevity of the benefits.

Results showed that almost all participants completed the study without experiencing serious side effects; most reported only mild soreness at injection sites. By the end of the treatment, participants gained an average of 4.5 pounds of lean muscle and shed approximately 3 pounds of fat. Notably, their walking distance improved by about 37 meters (120 feet), making daily activities like walking to the mailbox or navigating hallways noticeably easier.

Muscle strength increased approximately 3% beyond the expected range for their age and size. Participants also reported a reduction in overall disease burden, with many improvements persisting three months after stopping hormone injections.

These promising results pave the way for larger, controlled, multi-center trials to confirm the therapy’s efficacy in a broader population, including women. If future studies validate these findings, this hormone combination could become the first treatment to slow FSHD’s progression while helping patients regain lost strength and independence.

Experts believe this approach holds potential across various muscular dystrophies, especially considering the limited options for many rare neuromuscular diseases. The versatility of combination therapy could revolutionize treatment strategies for multiple underdiagnosed and undertreated conditions.

Source: https://medicalxpress.com/news/2025-09-hormone-combination-strength-function-fshd.html