NICE Favors Zolgensma Being Added to England’s Public Health Program
England’s National Institute for Health and Care Excellence (NICE) issued its final Zolgensma’s appraisal, recommending that the gene therapy be…
Articles by Marta Figueiredo, PhD
NICE Initially Against Adding Evrysdi to UK Public Health System
The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam)…
Stress-induced Protein May Serve as New SMA Biomarker in Infants
Blood levels of a stress-induced protein are significantly higher in young spinal muscular atrophy (SMA) patients and associate with…
SMN2 Copy Number and Rare Variant Influence SMA Severity
The number of copies of the “backup” SMN2 gene and a rare genetic variant in this gene influence disease severity…
SMA May Carry Higher Risk of Fluid Buildup in Brain, Study Finds
Spinal muscular atrophy (SMA) patients may at a fourfold greater risk of hydrocephalus, or abnormal accumulation of cerebrospinal fluid (CSF)…
Apitegromab Safely Counters Muscle Growth Suppressor, Phase 1 Trial Finds
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance…
Neurofilaments May Not Be Biomarker for Older Patients
The blood levels of neurofilaments — a marker of nerve cell damage — may serve as a disease onset and…
Warning of TMA, Rare Blood Disorder, Added to Zolgensma’s Safety Label
Novartis Gene Therapies has added thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — to the list…
Sex, SMN2 Copy Number Influence SMA Type 3 Age of Onset
Sex and the number of copies of the “backup” SMN2 gene influence both the age of symptom onset and the…
#AANAM – Zolgensma May Lead to Faster, Greater Gains Than Spinraza
Zolgensma may result in faster and greater functional improvements than Spinraza (nusinersen) in infants with spinal muscular atrophy…