I rolled up to the checkout counter, rosy cheeks beaming in delightful mimicry of the friendly-faced pansies beside me. The greenhouse air was thick and sweet like the morning sun’s hot breath in summertime. As my caregiver placed several cartons of sprightly lantanas beside the cash register,…

Birthdays, including my own awesome one last month, and other notable days — Mother’s Day, Memorial Day, school year’s end — dot the May calendar. June’s tally of memorable days is no slouch, either. My husband, Randy, and I married in June (47 years ago!), as did both…

Children with spinal muscular atrophy (SMA) who started taking Spinraza (nusinsersen) before symptoms appeared retained their ability to swallow after approximately four years, while youngsters with later-onset SMA walked farther with less fatigue. These results, from analyses performed on multiple ongoing clinical trials, were presented by Biogen,…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Meaningful further improvements in motor skills were evident with apitegromab treatment in later-onset spinal muscular atrophy (SMA) patients on maintenance doses of Spinraza (nusinersen), updated Phase 2 TOPAZ trial findings show. “The TOPAZ results show that apitegromab has promising potential to benefit the large portion of individuals with SMA who still…