How velmanase alfa works in children under 3 with alpha-mannosidosis

This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.

This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.

This trial tests a new gene therapy called Vesemnogene Lantuparvovec for people with spinal muscular atrophy (SMA). It is designed for patients in low- and middle-income countries who cannot access or did not respond to other treatments. The goal is to see if the therapy is safe and effective in real-world conditions.

This study looks at the long-term safety and how well the body accepts olipudase alfa therapy in children under 2 years old who have a certain type of ASMD. It helps understand if the treatment works safely over time.

This treatment program gives an early access enzyme therapy called tralesinidase alfa directly into the fluid around the brain (via a small pump) to help children with Sanfilippo syndrome type B. It aims to slow or stop the worsening of symptoms.

This early-stage study tests an IV gene therapy that delivers a working copy of an enzyme gene to help the body make beta-galactosidase in GM1 gangliosidosis. It may help slow or improve disease symptoms and looks closely at safety and how well the treatment works.