I had Mrs. Ruby Miner for spelling in the seventh grade. Besides spelling words, we defined, pronounced, and wrote them neatly. Stepping into Room 217 usually brought on a slight spell of jitters, even for this decent speller. I can only imagine the palpitations had I not been an attentive student. Over 50 years later, […]
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Spinraza (nusinersen), a treatment for spinal muscular atrophy (SMA) that helps improve motor function, was found to also improve lung function in a 10-year-old girl with advanced SMA type 2, a case report says. The report, “Nusinersen improved respiratory function in spinal muscular atrophy type 2,” was published in the journal Pediatrics International. Most people […]
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The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise billing occurs when patients unexpectedly receive bills […]
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The problem with disability pride is that it’s a little bit mythical. It’s not something you can describe or explain. It’s an enigma. A paradox. It shouldn’t exist, but it does, and the illogic of it — valuing something the world abhors — makes the concept seem impossible. You can’t understand disability pride unless you’ve […]
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Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, this “difficult” decision was based on the “rapid advancements in the SMA treatment landscape in recent years,” and the fact that branaplam would “not offer […]
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A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient trials, a study reported. The study, “Preclinical Safety Assessment and Toxicokinetics of Apitegromab, an Antibody Targeting Proforms of Myostatin for the Treatment of Muscle-Atrophying Disease,” […]
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I can still see the golden afternoon of my first tea party like it was only yesterday. I swung open the wooden cottage gate. In the center of a secret garden surrounded by dense evergreen hedgerows, a buffet table sat beneath the spotlight of the sun, awaiting guests. An elegant tablecloth in a rosy shade […]
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SadBaby, an organization that raises donations to support children with spinal muscular atrophy (SMA), is launching a crowdfunding platform. The project, based on digital currency donations, intends to gather funds from supporters and investors to help families financially with the costly treatments associated with SMA. “SadBaby is doing its bit to lend a helping hand […]
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AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For this reason, Alliance Rx noted in a […]
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