by Atrium Health Wake Forest Baptist Bolus administration of 2 mg/kg 3,4-DAP reverses clinical signs of botulism and prolongs survival. A Summary of experimental models. B Median ± interquartile ratio (IQR) toxic signs for rats given a single injection of vehicle (black) or 3,4-DAP (pink) at 28 h after challenge with 0.44 ng/kg BoNT/A (2.5 LD50 BoNT/A; n = 4 per group)....
Study is first to report beneficial effects of Nusinersen in adults with longstanding spinal muscular atrophy type 3 IOS PRESS Amsterdam, November 11, 2019 -A study published in the Journal of Neuromuscular Diseases presents the first evidence of mild improvement or stabilization of motor and respiratory function in adults with spinal muscular atrophy type 3...
By ED SILVERMAN @Pharmalot NOVEMBER 15, 2019 Last February, Barbara Moore switched to a new medicine to combat a rare neuromuscular disease that had plagued her for nearly a quarter of a century. She had not planned to do so, but Moore had no choice: An effective treatment she had been taking all those years...
Vickie Moored can walk again. Her words don’t slur, her vision isn’t blurred, and she no longer collapses every day. The 65-year-old from Elkton, Va., says she has her life back, thanks to the stunning efficacy of a cheap, but unapproved, chemical called 3,4-DAP. It’s a medication she would not have but for the largesse...
About 20,000 people in the United States are living with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The invariably fatal disease kills the nerve cells that control walking, eating and breathing. Few people survive more than three years after diagnosis. Robert Bucelli, MD, PhD, measures Gregory Easter’s neuromuscular function. Easter, who has...
Penn study shows that cholesterol drug may help Friedreich’s ataxia patients PHILADELPHIA – In preclinical studies using cell models that mimicked liver cells of patients with the rare disease Friedreich’s ataxia (FA), a widely used cholesterol-lowering drug increased a precursor of HDL (high-density lipoprotein), the “good cholesterol,” according to new research published in PLOS ONE from the...
