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Documents - Biomarin
Results:
4 Documents
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bilingue/bilingual
English
Francais
Keyword...
HCU - Homocystinuria
MSUD - Maple Syrup Urine Disorder
PKU - Phenylketonuria
TYR - Tyrosinemia Type
UCD - Urea Cycle Disorder
The situation of Palynziq in Quebec: On air with "Lagacé le matin"
(English)
Author(s):
Landry, Marika; PCU Blog, Lagacé le matin
Rating:
No Rating
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Link - French
PKU-SSIS - Adolescent
(English)
Author(s):
Clinical Outcomes Solutions (COS)
Rating:
No Rating
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Description:
The PKU-SSIS will help researchers understand the patient experience with PKU. To help develop the questionnaire, we would ask adolescents with PKU and their parents/guardians to participate in two online surveys.
Flyer
PKU-SSIS - Adult
(English)
Author(s):
Clinical Outcomes Solutions (COS)
Rating:
No Rating
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Description:
The PKU-SSIS will help researchers assess symptoms and impacts on daily living and understand the patient experience of living with PKU. If you are eligible, we will ask you to participate in two online surveys sent to you two weeks apart.
Flyer
Dr Francois Feillet discussing the final results of the KAMPER study reporting on the long-term safety and efficacy of Kuvan (BH4)
(English)
Author(s):
Feillet, Francois, JIMD - Journal of Inherited Metabolic Disease
Rating:
No Rating
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Description:
Dr Francois Feillet discussing the final results of the KAMPER study reporting on the long-term safety and efficacy of Kuvan (BH4) in patients with phenylalanine hydroxylase deficiency (phenylketonuria). Francois makes an excellent case for the use of this treatment in 'responders', and even some patients who don't respond so well, and explains a little of what might be coming for everyone else.
Podcast
