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Documents - RESEARCH - Treatments and Therapies 2
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CanPKU+ Submits Feedback on Draft Recommendation for Loargys (Publication)
Author(s): CanPKU+; Chute, Tanya Rating: No Rating   Read Reviews  
  CDAFeedback
CDA Sephiance Submission (Publication)
Author(s):  Rating: No Rating   Read Reviews  
Description: none
  PDF
Patient and Clinician Group Input - Pegzilarginase (TBC) (Publication)
Author(s): CDA submission Rating: No Rating   Read Reviews  
Description: https://www.cda-amc.ca/sites/default/files/DRR/2026/SR0923_pegzilarginase_Patient_Clinician.pdf
  PDF
Fact Sheet - Sephience - Sepiapterin (Publication)
Author(s): CanPKU+; Chute, Tanya Rating: No Rating   Read Reviews  
  English PDF
PKU and sepiapterin data with Suzanne Hollander, MS, RD, LDN (Publication)
Author(s): https://www.contemporarypediatrics.com/ Rating: No Rating   Read Reviews  
  Link  |  PDF
MSUD Research Newsletter Spring 25 (Publication)
Author(s): CanPKU+; Chute, Tanya Rating: No Rating   Read Reviews  
Description: Gene Therapy Liver Transplant
  PDF
Canada’s national pharmacare plan – boon or bane? (Publication)
Author(s): Rawson, Nigel; Adams, John Rating: No Rating   Read Reviews  
Description: National pharmacare, as proposed by the federal government, will not improve patient access to innovative medicines without major changes to current gatekeeping practices. Without deeper reforms, it could make drug access worse for patients. Canadians used to be proud of their “national medicare,” but health care across the country is now in crisis (Angus Reid Institute 2023). There’s a significant shortage of doctors, nurses, and hospital beds, several million Canadians have no family physician (Duong and Vogel 2023), emergency rooms are overrun with people who have nowhere else to turn for medical treatment (Kirkey 2024; Zafar 2024), and wait times to see a specialist (assuming an appropriate one is accessible in your area) and for surgery are far too long (Moir and Barua 2023) such that patients can die before seeing a health care provider.
  Link
The situation of Palynziq in Quebec: On air with "Lagacé le matin" (Publication)
Author(s): Landry, Marika; PCU Blog, Lagacé le matin Rating: No Rating   Read Reviews  
  PDF  |  Link - French
Alltrna presents preclinical data demonstration proof of concept for first trna development candidate (Publication)
Author(s): Alltrna Press Release on prnewswire Rating: No Rating   Read Reviews  
  Link
Dr Francois Feillet discussing the final results of the KAMPER study reporting on the long-term safety and efficacy of Kuvan (BH4) (Publication)
Author(s): Feillet, Francois, JIMD - Journal of Inherited Metabolic Disease Rating: No Rating   Read Reviews  
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
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