Quantitative MRI (qMRI) imaging of the muscles is a useful technique for identifying signs of compromised muscle health in people…
Articles by Lindsey Shapiro, PhD
Dysfunction of the phrenic nerve, which is important for controlling breathing, was observed in a small study of late-onset…
A powerful imaging technique called high-resolution intravital microscopy, or IVM, may be a useful tool for visualizing and quantifying the…
Infantile-onset Pompe disease (IOPD) patients who develop antibodies against enzyme replacement therapy (ERT) exhibit a distinct immune profile…
People with late-onset Pompe disease (LOPD) treated with Nexviazyme (avalglucosidase alfa) may be more than twice as likely…
More late-onset Pompe disease (LOPD) patients treated with Nexviazyme (avalglucosidase alfa) achieved clinically meaningful improvements in patient-reported outcome (PRO)…
A stem-cell based gene therapy that’s modified for better entry into muscle cells was seen to normalize protein levels in…
Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a combination therapy formerly known as AT-GAA, has been approved by the U.S.
Among children with classic infantile-onset Pompe disease (IOPD), enzyme replacement therapy (ERT) was associated with prolonged survival, but mortality…
Treatment with AT-GAA, Amicus Therapeutics’ experimental two-part therapy for Pompe disease, has now been shown to improve motor…