the secondary outcomes ICOAP intermittent and constant pain (statistically significant and clinically relevant differences between the groups at 3 months but not at 12 months follow-up), but not for the other outcomes (no differences between the groups at 3 months and at 12 months follow-up). No adverse events were reported during the study. Conclusions: At 3 months follow-up there was a statistical significant difference between the treatment groups in favor of the group allocated to exercise therapy on the primary outcomes HOOS pain and HOOS function and also in the secondary outcome ICOAP. At 12 months follow-up there were no statistical differences between the treatment groups.

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gina shaw N early 30 percent of infants with type 1 spinal muscular atrophy (SMA) were able to sit without support for at least five seconds after 12 months of treatment with risdiplam (Evrysdi, Genentech), the first oral therapy for SMA, according to results from a phase 2, openlabel study published in the New England Journal of Medicine on July 29."This is wonderful to see," said Seth Perlman, MD, medical director of the Neuromuscular Program at Seattle Children's Hospital, who was not involved with the study. "Sitting independently for any length of time is a milestone that just doesn't happen in babies with type 1 SMA without treatment. "Loss of motor function can occur rapidly in the first months of life for infants with type 1 SMA, Dr. Perlman said.Babies with type 1 SMA have no functional copies of the survival motor neu-ron1 (SMN1) gene, which encodes the survival motor neuron (SMN) protein. This protein is essential for the survival and proper functioning of spinal motor neurons. The severity of their disease is determined largely by their number of copies of the backup survival motor neu-ron2 (SMN2) gene, each of which can produce a small amount of SMN protein.

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