I’m getting ready for a trip to see my dear friend Christi get married this month. It’ll be the first time I meet her soon-to-be-husband in person, and from what she’s shared about him, I am so excited for that moment. Apparently, she has told him a lot about me…
Even when treated early with disease-modifying therapies (DMTs), infants and toddlers with spinal muscular atrophy (SMA) do not develop swallowing skills at the same pace or to the same level as healthy children. That’s according to a new study from Germany, which tracked more than 30 children with…
Discussion
Note: This column describes the author’s own experiences with Cymbalta (duloxetine). Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy. Every morning, my caregivers present me with a bouquet of drugs. What started as a measly two syringes has become a…
Genentech will not advance emugrobart (GYM329) into Phase 3 development, after early results from a clinical trial testing it in combination with Evrysdi (risdiplam) in people with spinal muscular atrophy (SMA). According to a letter to the SMA community, this “difficult decision” follows results from the first…
In 2018, when Nell Choi was 9 years old, she began experiencing symptoms that led to hospitalization and a diagnosis of neuromyelitis optica spectrum disorder (NMOSD), type positive. A month later, when Nell came home, she was weak, in a wheelchair, and had lost 20 pounds. To…
The motor function benefits of Itvisma (onasemnogene abeparvovec-brve), one of two Novartis’ gene therapies for spinal muscular atrophy (SMA), continued to increase over more than one year of treatment, extended Phase 3 trial results show. These findings come from the STEER Phase 3 trial (NCT05089656), which supported…
Get regular updates to your inbox.
