4 results on '"Deegan, Patrick B."'
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2. Lyso-Gb1 as a biomarker of the real-world situation in Gaucher disease: Comparative data from the Gaucher Outcome Survey (GOS) in ERT-treated and untreated patients
3. Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study
4. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study
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