Children walk after drug breakthrough

• Autores: Michael Le Page
• Localización: New scientist, ISSN 0262-4079, Nº. 3094, 2016, pág. 8
• Idioma: inglés
• Texto completo no disponible (Saber más ...)
• Resumen

  • Francesco Muntoni, at University College London, is talking about videos of children given an experimental drug for treating spinal muscular atrophy. This genetic disorder involves the deterioration of nerves connecting the brain and spinal cord to the body's muscles. Children with the severest form can't sit upright and seldom survive past the age of 2. Yet a few parents have posted videos online showing children given the drug, called nusinersen, who appear to be sitting and even walking with assistance. The trial of nusinersen was stopped in August when it became clear it was effective, making it unethical not to give the real drug to those on the placebo.