Oct 08, 2019
Gene therapy for Duchenne muscular dystrophy safely preserves muscle function
Study shows delivering a "distant cousin" of a key protein prevented muscle damage without triggering an immune response in large animal models.
Jun 14, 2019
Researchers’ discovery could lead to improved therapies for duchenne muscular dystrophy
Tiny protein could play a major role in combating heart failure related to Duchenne muscular dystrophy.
Jan 28, 2019
In myasthenia gravis, surgery to remove thymus gland provides benefits even years later
The surgery improves clinical outcomes for patients and may result in significant health care savings.
Jan 16, 2019
Pioneering surgery restores movement to children paralyzed by acute flaccid myelitis
An innovative and complex surgery involving nerve transfers is restoring hope and transforming lives torn apart by this mysterious and devastating illness.
Aug 30, 2018
Obstructive sleep apnea linked with higher risk of gout
People with obstructive sleep apnea have higher risk of developing gout, even beyond first years after being diagnosed with the sleep disorder.
Aug 29, 2018
Spinal muscular atrophy drug may be effective if started later than previously shown
Drug shown to be effective in treatment of babies with spinal muscular atrophy may be effective for muscle control even when treatment is started in children 7 months and older.
Mar 27, 2018
Preclinical testing suggests some antioxidants may be effective in treating mitochondrial disease
Researchers test 7 compounds in 3 model systems, seeking leads for clinical trials.
Mar 21, 2018
Tamoxifen and raloxifene slow down the progression of muscular dystrophy
Long-term treatment with selective estrogen receptor modulators (SERMs) improves muscle, respiratory, and skeletal function without weakening bone in new mouse studies.
Dec 11, 2017
Drug for spinal muscular atrophy prompts ethical dilemmas
First drug for people with spinal muscular atrophy gives hope for improving the lives of patients with the rare disease, but the extraordinary cost of the drug and complicated logistics of delivering it present barriers for many patients.