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.
Sep 26, 2017
Restoring breathing capacity in Duchenne muscular dystrophy by activating the brain
Enhancing breathing via the brain may limit deficiencies in respiratory capacity in Duchenne muscular dystrophy patients.
Jul 14, 2017
Patients receiving eculizumab at high risk for invasive meningococcal disease despite vaccination
Some patients receiving eculizumab who were vaccinated with the recommended meningococcal vaccines still developed meningococcal disease. Of important note, eculizumab is currently under US FDA review as potential treatment for patients with generalized myasthenia gravis who are AChR antibody-positive.
Jun 28, 2017
Designed proteins to treat muscular dystrophy
In an animal mode of congenital muscular dystrophy researchers demonstrated for the first time that 2 proteins not only recover muscle force and increase body weight in the sick animals but also significantly prolong survival.The study findings are published in Science Translational Medicine.