SMA News Today

June 20, 2022June 20, 2022

MicroRNAs in Blood May Predict Spinzara Response in Type 1 Patients

SMA News Today

MicroRNAs isolated from the bloodstream of patients with infant-onset spinal muscular atrophy (SMA) type 1 before treatment were related to functional responses to Spinraza (nusinersen) after six months of therapy, ... Read more

June 17, 2022June 17, 2022

Neurologists Confident SMA Treatments Help Youngest Patients Most

SMA News Today

Pediatric neurologists’ expectations in terms of treatment benefits are decidedly higher for infants and toddlers with spinal muscular atrophy (SMA) types 1 and 2 than for adolescent patients, a study ... Read more

June 15, 2022June 15, 2022

Medical Costs Lower if Treatment Is Started Before Symptoms: Study

SMA News Today

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 ... Read more

June 13, 2022June 13, 2022

Manitoba Newborn Screening Program Expands to Include SMA

SMA News Today

The newborn screening program at Cadham Provincial Laboratory in Manitoba, Canada, is expanding to include spinal muscular atrophy (SMA), according to an announcement from Audrey Gordon, the province’s health minister. ... Read more

June 10, 2022June 10, 2022

Higher Spinraza Dose May Be More Effective, Modeling Study Predicts

SMA News Today

A new modeling study predicts that higher doses of Spinraza (nusinersen) may improve its effectiveness compared with the approved 12 mg dose. The study used data from two clinical trials ... Read more

June 8, 2022June 8, 2022

Motor Function Linked to Cognitive Differences for SMA Type 3 Adults

SMA News Today

Among adults with spinal muscular atrophy (SMA) type 3, those with greater motor impairment tend to score better on some cognitive measures, but worse on others, a new study reports. ... Read more

June 6, 2022June 6, 2022

Combining Evaluation Tools May Detect Early Signs of SMA: Study

SMA News Today

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 ... Read more

June 3, 2022June 3, 2022

Infants Screened for Zolgensma Should Repeat Antibody Tests

SMA News Today

Infants with spinal muscular atrophy (SMA) who are being screened for Zolgensma may present with maternal antibodies against the therapy’s viral carrier — making them ineligible for the therapy — until ... Read more

June 1, 2022June 1, 2022

Evrysdi Gets FDA Approval for SMA Babies Under 2 Months Old

SMA News Today

The U.S. Food and Drug Administration (FDA) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old. Evrysdi, an oral ... Read more

May 27, 2022May 27, 2022

MicroRNAs Can Predict Response to Spinraza, Study Suggests

SMA News Today

MicroRNAs isolated from people with spinal muscular atrophy (SMA) types 2 and 3 before treatment predicted eventual responses to Spinraza (nusinersen) therapy, a study suggested. The study, “Muscle microRNAs in ... Read more

May 25, 2022May 25, 2022

UK Collaboration Gets £1.25M Grant to Develop ‘Smart Suit’

SMA News Today

Development of an exoskeleton suit to help those living with progressive neuromuscular conditions that affect their upper-body movement will advance due to a £1.25 million (about $1.5 million) grant from ... Read more

May 23, 2022May 23, 2022

CADTH Panel Recommends Against Reimbursing Spinraza for Adults

SMA News Today

A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with ... Read more

May 20, 2022May 20, 2022

Evrysdi Safe in Type 1, 2 Patients in US Access Program Before Approval

COVID-19 News, SMA News Today

Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical ... Read more

May 18, 2022May 18, 2022

Zolgensma Safe in Children Heavier Than Those in Trials, Data Show

SMA News Today

Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier ... Read more

May 16, 2022May 16, 2022

From Actor to Attorney, Woman’s ‘Work Ethic Outshines My Disability’

SMA News Today

From being a child actor to a lawyer in the entertainment industry, Alexa Dectis, who has relied on a power wheelchair to get around since she was 2, has gone ... Read more