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  • less
  • With less than 20% of wheelchair and walker users being employed, it's becoming a bit of a phenomenon to change the way disabilities affect our lives. (caregiverproducts.com)
  • motor
  • Since SMA results from deficiency of the Survival Motor Neuron (SMN) protein, putting SMN back in motoneurons is thought to be able to reverse motoneuron loss and therefore the disease. (nhsjs.com)
  • patients
  • While we have far greater uncertainties about the exact net benefits of Zolgensma than nusinersen, the magnitude of effect in these…patients was large enough to have high certainty that Zolgensma provides a substantial net health benefit compared with standard care," the report states. (xconomy.com)
  • Most notably, when the BBB was generated from cell samples of patients with HD, the BBB malfunctioned in the same way that it does in patients with the disease. (ca.gov)
  • months
  • Spinraza, which is administered by spinal injection every four months, has a U.S. list price of $750,000 (£581,122) for the first year and $375,000 (£290,561) annually thereafter. (pharmalive.com)
  • The first signs of the disease appeared in Maisy when she was just 10 months old and at 15 months she was diagnosed with the condition. (omar.co.uk)
  • drug
  • The drug is approved for all forms of SMA, and has shown an ability to potentially change the trajectory of the disease, particularly when administered early . (xconomy.com)
  • Boston Children's Hospital is suing a Saudi prince it says has reneged on a commitment to pay for the care of a baby with a rare disease that can only be treated with an incredibly expensive drug. (universalhub.com)
  • physical
  • As a result, this is a devastating disease that exerts tremendous pain, both physical and psychological, on affected individuals and their families. (nhsjs.com)
  • world
  • The doctor did mention some clinical trails that were taking place so I just emailed probably every doctor linked to Spinal Muscular Atrophy around the world to find out where the trials were and if there was any chance of getting him on them. (samebutdifferentcic.org.uk)
  • time
  • This allowed the researchers to create what is called a "disease-in-a-dish" model that allowed them to see, in real time, what is happening in AMD. (ca.gov)