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The introduction of Spinraza (nusinersen) as a treatment for spinal muscular atrophy (SMA) in Sweden was associated with trends of improved survival in children diagnosed with the disease before they were 2 years old, a nationwide population-based study shows. While these trends did not reach statistical significance…
In its final decision, the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended against Spinraza (nusinersen) being reimbursed to treat adults with spinal muscular atrophy (SMA). The decision was met with dismay by the SMA community and Biogen, Spinraza’s manufacturer. “We are absolutely devastated for…
Prescribing information for Zolgensma (onasemnogene abeparvovec-xioi), an approved gene therapy for spinal muscular atrophy (SMA), is being updated following the death of two patients who developed acute liver failure after treatment. Acute liver failure was known to be a potential severe side effect of Zolgensma, highlighted in…
Photo courtesy of Jessica Keogh Day 28 of 31 This is Jessica Keogh‘s story: I’m Jessica Keogh, and I’m the oldest of three, but the only one who shows physical symptoms of spinal muscular atrophy with respiratory distress, or SMARD.
Photo courtesy of Maxwell McKnight Day 27 of 31 This is Maxwell McKnight’s (@maxwellmnight) story: Hello! My name is Maxwell. I’m 18 and have just finished my A levels. I have spinal muscular atrophy (SMA) type 2. This September, I begin my journey into…
The sense of physical fatigue and perceived fatigability, or the impact of reduced physical performance on daily life activities, are frequent and disabling symptoms in adults with spinal muscular atrophy (SMA) at different functional levels, a study showed. Such symptoms — whose frequency was generally associated with motor impairment —…
Doctors expected Stephen Mikita to live for six months after his diagnosis, in the late 1950s, with spinal muscular atrophy (SMA) type 2. Now 66, Mikita can look back on a successful career in law, first as a law clerk to the late U.S. Sen. Orrin Hatch, a…
Kevin,….Way to go…..terrific article…….
The relationship between a physical therapist and patient is very important for many reasons and should be an open, honest, and trusting relationship. This is especially important when you are working with someone with SMA, or another neurological disorder, who you will most likely be seeing long-term. It is important…
Newborn screening for spinal muscular atrophy (SMA) leads to better outcomes, with babies being diagnosed earlier and with prompt access to life-saving treatments, according to three-year data from the SMA screening program in the state of New York. Gene therapy was the main treatment strategy, and delays in…
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