Major Developmental Milestones Met in Infants, Zolgensma Data Show
New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before...
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a...
Safety For Both Therapist and SMA Patient
While working with a patient with SMA, you may find it beneficial for the patient, as well as the therapist, to have at least two people available to help in the treatment if you plan to transfer to a therapy table for different exercises. For Michael, we transfer him...
Newborn Screening in Massachusetts Seen as Highly Accurate, Effective
Newborn screening (NBS) for spinal muscular atrophy (SMA) in Massachusetts allowed for early detection and treatment of affected infants, with most referred to a specialist within the first week of life, three-year data from the state’s SMA screening program show....
SMA News Today Launches Survey to Gain Understanding of Disease Community
SMA News Today is conducting a U.S.-based survey to better understand the characteristics, needs, and treatment experiences of the spinal muscular atrophy (SMA) community. Adults — from patients to clinicians — ages 18 and older are invited to participate. The survey...
#CureSMA2021 – Spinraza Leads to Better Swallowing, Farther Walking
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...
Get Ready for NORD’s Patient and Family Forum, Set for June 26-27
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...
#CureSMA2021 – Apitegromab Safely Increases Motor Abilities in SMA Types 2, 3
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...
#CureSMA2021 – Evrysdi Linked to Improved Motor Function in 2 Trials
Evrysdi (risdiplam) continues to be linked to motor function improvements in a broad range of people with spinal muscular atrophy (SMA), according to recent data from two ongoing Phase 2 clinical trials. These data were presented at the 2021 Virtual SMA Research &...
#CureSMA2021 – Spinal Catheter Safe and Effective in Using Spinraza
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 Virtual SMA Research & Clinical Care Meeting, held June 9–11. Go here to read stories from the conference. Delivering Spinraza (nusinersen) directly into the upper spinal cord...HCP — SMA News Today
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