Expanded access to velmanase alfa for alpha-mannosidosis

This study looks at how the drug velmanase alfa (Lamzede) works in the body of young children with alpha-mannosidosis. It's for children who started the drug before age 3.

This expanded access program provides migalastat (a pill) to people with Fabry disease who have a specific genetic mutation and cannot use standard enzyme replacement therapy. It's an option for those who need treatment and have no other alternatives.

This program provides early access to an experimental enzyme therapy given directly into the brain for children with Sanfilippo syndrome type B, a genetic condition that affects the nervous system. The treatment aims to replace a missing enzyme to slow or stabilize symptoms.

This trial tests a treatment called velaglucerase beta, which replaces an enzyme that people with Gaucher disease lack. It may help reduce enlarged spleen and liver, improve anemia, and increase platelet counts in children under 12.

This program provides access to Mepsevii, an enzyme replacement therapy, for people with MPS VII who cannot get the drug through their regular healthcare system. It is a way to try the treatment outside of a clinical trial.

This trial provides access to an investigational combination therapy for Pompe disease when standard treatments are not suitable. It aims to help people with Pompe disease who cannot join other clinical studies or are no longer benefiting from current enzyme replacement therapy.