Leah Zelaya, a 15-year-old with a rare form of spinal muscular atrophy (SMA), was chosen by the Muscular Dystrophy Association (MDA) to be its 2023 MDA National Ambassador. As national ambassador, the Brooklyn, New York, teenager represents all those living with a neurological disease in the U.S. She and…

The team at SMA News Today brought you daily coverage of the latest developments in treatment and advancements in research related to spinal muscular atrophy (SMA) in 2022. We look forward to continuing to serve the SMA community in the new year. Here we’ve compiled a list of…

Without disease-modifying treatment (DMT), spinal muscular atrophy (SMA) carries high mortality rates and steep healthcare costs, a study in Hong Kong reported. Not surprisingly, the greatest loss of life and highest costs were observed in children with SMA type 1, one of this disease’s most severe forms with…

The incidence of infantile spinal muscular atrophy (SMA) has increased in Japan since 2016 and this could be related to greater disease awareness among clinicians, earlier diagnoses, and the introduction of new treatments — although this relationship remains to be established, a study has found. On the large Japanese…

From left, Corey, Kate, and John Weber. (Photo courtesy of the Weber Family) Day 26 of 31 This is John, Corey, and Kate Weber’s story: When my husband John and I learned our 4-month-old daughter, Kate, had SMA type 1, some of our first thoughts…

Kristen and her son Jack. (Photo courtesy of Kristen Resendez) Day 11 of 31 This is Kristen Resendez’s story: As a mom, my favorite time of the year is the Cure SMA Annual Conference. It’s the only time of the year when I know my son can be…

Spinraza (nusinersen) treatment was generally well tolerated and led to improved or stabilized motor function for nearly 300 children with spinal muscular atrophy (SMA) in Poland, a new study reports. The study, “Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular…

Zolgensma safely and effectively halts disease progression in infants genetically diagnosed with spinal muscular atrophy (SMA) but not yet showing symptoms, according to final data from the SPR1NT Phase 3 clinical trial. “When treated with Zolgensma prior to the onset of symptoms, not only did all 29 patients…

The total medical costs associated with spinal muscular atrophy (SMA) are lower for patients diagnosed at birth via newborn screening, compared with those diagnosed after they start showing symptoms of the disease, a new study has found. These findings “demonstrate clearly that as long as the decision to reimburse…

Combining two clinical assessment tools helped recognize early neurological signs in infants with spinal muscular atrophy (SMA) who were identified through a newborn screening program, a study suggested. Even in the absence of obvious clinical signs, infants typically defined as presymptomatic may have minimal signs, such as poor muscle…