Rare Disease Day 2023
Recent Posts
- Same mutation caused late-onset and classic Fabry in same family
- Genetic testing recommended for family members of Fabry patients
- EC decision due in May on enzyme replacement therapy PRX-102
- Impairment in nerves that regulate heartbeat common in Fabry disease
- Galafold may reduce heart changes, boost exercise tolerance
- Lymphedema affects more than 15% of patients; screening recommended
- FDA puts clinical hold on Fabry disease gene therapy 4D-310
- Changes in Heart, Kidney Function May Be Undiagnosed Fabry Disease
- Early Data for Gene Therapy FLT190 Support Ongoing MARVEL-1 Trial
- Galafold Adherence High as Patients Report Life Quality Gains