Sandra Sirrs

Transplantation as disease modifying therapy in the era of gene therapy medicinal products – health policy considerations

Margreet Wagenmakers, Anna Lehman, Caroline M. den Hoed, Laura van Dussen et autres

BACKGROUND: New gene therapy medicinal products [GTMP] are being considered as alternatives to liver transplant [LTx] for some patients with inherited metabolic diseases [IMDs] but pose unique challenges for health policy makers. METHODS: Published data on LTx and GTMP in human patients …

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Treatment goals for adults with early treated PKU should be determined by evidence-based shared decision making between patients and their medical team

Mirjam Langeveld, Sandra Sirrs, Robin Lachman, Timothy Fazio et autres

This opinion article focusses on treatment targets in adults with early treated PKU (etPKU), specifically the recommended phenylalanine (Phe) level. The current body of evidence on the relationship between Phe levels at different times in life and neurocognitive functioning and quality of …

nl, ca, gb, au, de, hu, ch, se (code pays fourni par la source)

Additional file 1 of Treatment goals for adults with early treated PKU should be determined by evidence-based shared decision making between patients and their medical team

Mirjam Langeveld, Sandra Sirrs, Robin Lachman, Timothy Fazio et autres

Supplementary Material 1

nl, ca, gb, au, de, hu, ch, se (code pays fourni par la source)

Treatment goals for adults with early treated PKU should be determined by evidence-based shared decision making between patients and their medical team

Mirjam Langeveld, Sandra Sirrs, Robin Lachman, Timothy Fazio et autres

Abstract This opinion article focusses on treatment targets in adults with early treated PKU (etPKU), specifically the recommended phenylalanine (Phe) level. The current body of evidence on the relationship between Phe levels at different times in life and neurocognitive functioning and quality …

nl, ca, gb, au, de, hu, ch, se (code pays fourni par la source)

Additional file 1 of Treatment goals for adults with early treated PKU should be determined by evidence-based shared decision making between patients and their medical team

Mirjam Langeveld, Sandra Sirrs, Robin Lachman, Timothy Fazio et autres

Supplementary Material 1

nl, ca, gb, au, de, hu, ch, se (code pays fourni par la source)

Treatment goals for adults with early treated PKU should be determined by evidence-based shared decision making between patients and their medical team

Mirjam Langeveld, Sandra Sirrs, Robin Lachman, Timothy Fazio et autres

Abstract This opinion article focusses on treatment targets in adults with early treated PKU (etPKU), specifically the recommended phenylalanine (Phe) level. The current body of evidence on the relationship between Phe levels at different times in life and neurocognitive functioning and quality …

nl, ca, gb, au, de, hu, ch, se (code pays fourni par la source)

Lost to Follow-Up: The Challenges of Maintaining Contact Through the Lifespan

Sandra Sirrs, Anna Lehman, Graeme A. M. Nimmo

ca (code pays fourni par la source)

Oral Micronized Progesterone for Perimenopausal Vasomotor Symptoms Study

Prior, Jerilynn C., 1943-, Andrea Cameron, Michelle Fung, Christine L. Hitchcock et autres

This is a randomized, double-masked, placebo-controlled trial of oral micronized progesterone (300 mg at bedtime daily) for hot flushes and night sweats (collectively vasomotor symptoms, VMS) in women stratified to be in Early or Late Perimenopause. We have recently completed a similar …

ca (code pays fourni par la source)