Newsletter, MARCH 2021

  30th March 2021 Newsletter March 2021 The recent approval by regulatory agencies worldwide of Zolgensma, a gene therapy for children with type 1 Spinal Muscular Atrophy 1 (SMA1), raised happiness and gratitude across families and scientific communities. SMA1 is a genetic neuromuscular disorder that affects the nerve cells that control voluntary muscles (motor neurons). [...]