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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