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One of the first objectives we wanted to address when Michael, who has spinal muscular atrophy (SMA), began physical therapy in 2017, was swelling in his feet and ankles. There are many ways we have addressed the swelling and edema in his legs, including reducing his salt intake.
The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic infants in England who are genetically predisposed to spinal muscular atrophy (SMA). Per updated recommendations from the country’s National Institute for Health and Care Excellence (NICE), this…
High doses of Evrysdi (risdiplam), an approved daily therapy for spinal muscular atrophy (SMA), affected the development of sperm in rats and monkeys, a review of 14 preclinical safety studies reported. No damage was evident in immature sperm derived from stem cells, and changes affecting sperm ended…
Evaluating the strength of nerve cell impulses to muscles may be a good way of predicting motor skill recovery after Zolgensma (onasemnogene abeparvovec) treatment in infants with spinal muscular atrophy (SMA), a recent study suggests. The research showed that compound muscle action potential (CMAP) amplitudes in the…
A common complaint SMA patients have is that their feet swell due to a lack of circulation, as well as being seated in a wheelchair most of the day. In this position, gravity allows for the pooling of fluid in the feet. Since there isn’t much, if any,…
Previously, we have performed cupping and dry needling on different parts of Michael’s body to help with several ailments throughout his time in physical therapy. Recently, cupping has helped reduce pain and increase the range of motion in his cervical spine, or neck. Michael reported after cupping…
In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the company is giving Royalty Pharma a percentage of the payments it receives from sales of Spinraza (nusinersen), an approved therapy for spinal muscular atrophy (SMA). In exchange, …
The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy (SMA) is expected to complete patient enrollment later this year, the company announced. Top-line data from the trial are anticipated in 2024. Should results be positive, Scholar Rock…
CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for spinal muscular atrophy (SMA). The company had completed preclinical studies of the investigational gene therapy in collaboration with the Horae Gene Therapy Center at the University of Massachusetts (UMass)…
BraunAbility has partnered with the nonprofit organization Laughing at My Nightmare (LAMN) to donate a wheelchair-accessible vehicle to a 6-year-old girl with spinal muscular atrophy (SMA). Fynlee Armstrong, a “sassy and spunky little girl” from Clyde, Texas, requires a wheelchair to move around. Over the last few…
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