Altered gut bacteria may contribute to metabolic disorders in SMA: Study
Alterations to the composition of intestinal microbes — called the gut microbiome — in spinal muscular atrophy (SMA) patients…
Articles by Lindsey Shapiro, PhD
Genetic sequencing assay for rare disease diagnosis launched
Centogene has launched a new genetic sequencing assay to support faster and more accurate diagnoses of rare diseases, such as…
Akron Children’s earns $1.3M to improve pediatric palliative care
Akron Children’s Hospital in Ohio has been awarded $1.3 million to optimize palliative care practices for children living with chronic…
CANbridge will seek FDA OK for human trials of new gene therapy
CANbridge Pharmaceuticals is planning to ask the U.S. Food and Drug Administration for permission to launch, by the end…
MDA 2023: Evrysdi may work better than Spinraza for SMA type 1
Evrysdi (risdiplam) may be associated with longer survival and more motor function gains than Spinraza (nusinersen) in children…
MDA 2023: Zolgensma may benefit patients with more SMN2 copies
Most spinal muscular atrophy (SMA) patients with four or more copies of the backup SMN2 gene achieved or maintained motor…
MDA 2023: Apitegromab leads to sustained gains for some patients
Treatment with apitegromab for two years continues to stabilize or improve measures of motor function, fatigue, and life quality…
MDA 2023: Evrysdi’s motor benefits found to last for 4 years in trial
Motor function is still stable or improved among people with spinal muscular atrophy (SMA) types 2 and 3 after…
Zolgensma gene therapy for SMA now routinely accessible in UK
The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic…
Nerve impulse test seen to predict motor recovery after Zolgensma
Evaluating the strength of nerve cell impulses to muscles may be a good way of predicting motor skill recovery after…