Autori: Goemans N

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Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy

Clinical Variability in Spinal Muscular Atrophy Type III

Gain and loss of abilities in type II SMA: A 12-month natural history study

Age and baseline values predict 12 and 24-month functional changes in type 2 SMA

Performance of Upper Limb module for Duchenne muscular dystrophy

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