News – SMA News Today The Web's Daily Resource for Spinal Muscular Atrophy News
- NICE Initially Against Adding Evrysdi to UK Public Health Systemby Marta Figueiredo PhD on June 4, 2021
The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam) to the list of medications available to spinal muscular atrophy (SMA) patients through […]
- Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hillby Patricia Inacio PhD on June 2, 2021
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners […]
- Asuragen’s Lab Test Can Speed Up Diagnosis of SMA Carriers, Patientsby Patricia Inacio PhD on June 2, 2021
Asuragen’s lab test for spinal muscular atrophy (SMA) is as robust and accurate at identifying carriers of the disease and diagnosing patients as more traditional methods, a study shows. The diagnostic test — called AmplideX PCR/CE SMA Plus […]
- Stress-induced Protein May Serve as New SMA Biomarker in Infantsby Marta Figueiredo PhD on May 28, 2021
Blood levels of a stress-induced protein are significantly higher in young spinal muscular atrophy (SMA) patients and associate with levels of a nerve cell damage biomarker, a study shows. These findings from untreated SMA patients suggest that this protein, […]
- Muscle-directed Therapy Apitegromab Put on FDA’s Fast Trackby Patricia Inacio PhD on May 26, 2021
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was granted a fast track designation by the U.S. Food and Drug Administration. “We are delighted to receive Fast Track designation and look forward to working […]
- SMN2 Copy Number and Rare Variant Influence SMA Severityby Marta Figueiredo PhD on May 24, 2021
The number of copies of the “backup” SMN2 gene and a rare genetic variant in this gene influence disease severity in people with spinal muscular atrophy (SMA), according to data from more than 2,000 people involved in a free SMA genetic testing […]
- SMA May Carry Higher Risk of Fluid Buildup in Brain, Study Findsby Marta Figueiredo PhD on May 21, 2021
Spinal muscular atrophy (SMA) patients may at a fourfold greater risk of hydrocephalus, or abnormal accumulation of cerebrospinal fluid (CSF) in the brain, than their healthy peers, a large U.S. health record-based study suggests. Data also showed that […]
- Apitegromab Safely Counters Muscle Growth Suppressor, Phase 1 Trial Findsby Marta Figueiredo PhD on May 19, 2021
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance of the inactive form of myostatin, a muscle growth suppressor, in healthy volunteers, […]
- Cure SMA Offers US Patients Free Tools to Boost Their Independenceby Mary Chapman on May 17, 2021
Cure SMA is offering free “independence assistance packages” to teenagers and adults with spinal muscular atrophy (SMA) in the U.S. to assist with everyday living or to help make living on their own possible. The Teen & Adult Independence […]
- Neurofilaments May Not Be Biomarker for Older Patientsby Marta Figueiredo PhD on May 12, 2021
The blood levels of neurofilaments — a marker of nerve cell damage — may serve as a disease onset and treatment response biomarker in infants with spinal muscular atrophy (SMA) type 1, but not in older patients with milder disease forms, […]
