Side effects

Hypersensitive reactions to infusions of enzyme replacement therapies (ERT) to treat late-onset Pompe disease (LOPD) were primarily mild and nonallergic, a small study reported. Many reactions were reported after a year of well tolerated ERT and most of them occurred in women, data showed. After the reactions, the ERT…

No anesthesia complications were reported and surgery was successful for a man with late-onset Pompe disease and airway problems who broke a bone in his upper arm, according to a case report from Spain. The man, 47, underwent surgery for the broken left arm under a combined anesthesia approach after…

Anaphylaxis, a severe allergic reaction, occurred in nearly one-third of children with Pompe disease treated with the enzyme replacement therapy Myozyme (alglucosidase alfa) in a small study. However, rapid desensitization regimens — using small doses initially and then increasing the doses — safely re-established tolerance to Myozyme in…

Acceptable safety is being seen to date in four adults with late-onset Pompe disease (LOPD) given the one-time gene therapy  AT845 in the FORTIS Phase 1/2 trial, its developer, Astellas Gene Therapies, announced. Interim study data in these four enrolled patients, as of the Dec. 3 cutoff date, shows…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

People with Pompe disease will likely need, at some point, to undergo medical procedures that require anesthesia. Here is information about the different forms of anesthesia, how Pompe disease may complicate its use, and precautions doctors should take. What is Pompe disease? Pompe is a rare genetic disease caused…

Patients with Pompe disease may have to undergo surgery requiring general anesthesia. Below is information about the disease, why patients may need surgery, and the potential risks of anesthesia. What is Pompe disease? Pompe disease is a rare genetic disease caused by a mutation in the GAA…

Children receiving enzyme replacement therapy (ERT) for Pompe disease often experience immune reactions to treatment, presenting as rashes or severe allergy, sometimes after several successful infusions, a case series shows. While these hypersensitivity reactions can be avoided successfully with anti-allergy medications and longer infusion times in some patients, others…

Low-dose treatment with methotrexate can reduce the immune reaction that often accompanies enzyme replacement therapies in infants with Pompe disease, researchers report. Their study, “An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe…

Adding immunomodulatory medicines to enzyme replacement therapy (ERT) — Myozyme (alglucosidase alfa) — gives temporary but not long-term protection from antibodies that can block ERT’s effectiveness in patients with classic infantile Pompe disease. Better immunomodulatory regimens are needed to induce long-term…