I don’t currently have access to live browsing for “latest news” updates right now, so I can’t reliably tell you what broke most recently this week/month. If you want, paste any links or headlines you’ve found and I’ll summarize and assess them.
That said, here are recently important, widely covered developments in glycogen storage disease type II (GSD II, Pompe disease) therapy and context that may help you interpret newer news:
Therapy approvals (context for “what’s new”)
- Avalglucosidase alfa (Nexviazyme) has been authorized/approved in major regions: EMA recommended authorization in July 2021, US approval in August 2021, and EU approval in June 2022.[5]
- Cipaglucosidase alfa: EMA recommended authorization in December 2022, with approval in June 2023 (noted as available across EU age groups without weight restrictions).[5]
What GSD II is (why treatment coverage keeps evolving)
- GSD II (Pompe disease) is caused by deficient acid alpha-glucosidase (GAA) activity leading to lysosomal glycogen accumulation; it’s classically described as a spectrum with severe infantile and later-onset forms.[7][8]
- Enzyme replacement therapy (ERT) has shown encouraging results particularly in infantile disease, while effectiveness in late-onset disease has been harder to pin down because outcomes vary and natural history is heterogeneous.[2][8]
If you tell me what “latest” means…
Reply with one of these, and I’ll tailor a “most recent” brief accordingly (or summarize the items you paste):
- “Last 7 days” / “Last month” / “Last year”
- Whether you mean clinical trials, drug approvals/regulatory actions, or patient/community news
If you paste 3–10 headlines/links, I’ll produce a clean summary with: what changed, who it affects (infantile vs late-onset), and why it matters.
Sources
News and Articles Updates of interest to the GSD community Latest News & Articles Past News & Articles
agsdus.orgClinical and genetic mutation analysis was performed on 5 infantile glycogen storage disease type II children in Chinese mainland. Clinical data of 5 children with infantile-type glycogen storage disease type II due to the acidic α-glucosidase (GAA) ...
pmc.ncbi.nlm.nih.govMethods of treating glycogen storage disease type II, by administering acid α-glucosidase, are described, as are compositions for use in treatment of glycogen storage disease type II.
patents.google.comGlycogen storage disease type II has a broad continuous clinical spectrum in terms of onset, involvement of organs and life expectancy. Infantile onset is the most severe form, presenting with prominent cardiomyopathy, hypotonia, hepatomegaly and ...
pmc.ncbi.nlm.nih.govGlycogen storage disease type II, also known as Pompe disease, is a rare and progressive neuromuscular disorder inherited in an autosomal recessive manner. This disease results from a deficiency of the enzyme acid α-glucosidase (GAA), causing impairment in the degradation of glycogen within the lyso …
pubmed.ncbi.nlm.nih.govGlycogen storage disease type II (GSDII) is an autosomal recessive lysosomal disorder caused by mutations in the gene encoding alpha-glucosidase (GAA). The disease can be clinically classified into three types: a severe infantile form, a juvenile ...
pmc.ncbi.nlm.nih.govGlycogen storage disease type II (GSDII) is an autosomal recessive lysosomal disorder caused by mutations in the gene encoding alpha-glucosidase (GAA). The disease can be clinically classified into three types: a severe infantile form, a juvenile and an adultonset form. Cases with juvenile or adult …
pubmed.ncbi.nlm.nih.govGlycogen storage disease type II (GSDII) is an autosomal recessive lysosomal disorder caused by mutations in the gene encoding alpha-glucosidase (GAA). The dise...
journals.sagepub.comGlycogen storage disease type II (GSD II) is characterized clinically by severe muscle weakness, moderate hepatomegaly, and substantial cardiac enlargement in an infant who appeared "healthy" at birth (see page 622). Hypotonia and cardiomegaly are so extreme that they cannot be missed. This ease of...
jamanetwork.com