archive-com.com » COM » P » POMPECANADA.COM

Total: 118

Choose link from "Titles, links and description words view":

Or switch to "Titles and links view".
  • New Brunswick Announces Plan to Treat Patients - Canadian Association of Pompe
    criteria The plan will cover the cost of five drugs for specific rare diseases Aldurazyme for the treatment of Hurler and Hurler Scheie forms of Mucopolysaccharidosis I Elaprase for the treatment of Hunter Syndrome Ilaris for the treatment of Cryopyrin Associated Periodic Syndrome Myozyme for the treatment of Pompe disease and Zavesca for the treatment of Niemann Pick Type C Source Govt of New Brunswick Tagged ERT getting treated Share this Facebook Twitter Google LinkedIn Related Recent Posts Inequities Between Provinces in Treating Rare Diseases Rare Disease Strategy Urgently Needed to Help 3 Million Canadians BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J P Morgan Annual Healthcare Conference Sanofi Genzyme Announces Recipients of Patient Advocacy Leadership PAL Awards Amicus Therapeutics Commences Phase 1 2 Study of Novel ERT for Treatment of Pompe Disease Categories Awareness CAP News Education Events Interviews Living with Pompe News Press Releases Research Studies Tags advocacy awards Awareness CAP chaperone CORD eLearning Emma Rooney ERT Extraordinary Measures Fabry fundraising Gaucher gene therapy getting treated grants guest blogger Int l Pompe Day IPA late onset living with Pompe meetings Muscular Dystrophy Canada MyNormal newborn screening Orphan Drug Framework Orphanet politics

    Original URL path: http://www.pompecanada.com/blog/news/press-releases/new-brunswick-announces-plan-to-treat-patients/ (2016-02-13)
    Open archived version from archive


  • News Archives - Canadian Association of Pompe
    Leadership PAL awards The global grant program supports projects by non profit organizations that serve patients living with lysosomal storage disorders LSDs a group of rare inherited disorders that can cause progressive and debilitating health problems Amicus Therapeutics Commences Phase 1 2 Study of Novel ERT for Treatment of Pompe Disease by CAP Dec 22 2015 Press Releases Amicus Therapeutics Nasdaq FOLD a biopharmaceutical company at the forefront of rare and orphan diseases today announced that its investigational new drug IND application submitted to U S Food and Drug Administration FDA is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1 2 study of ATB200 in patients with Pompe disease Ten Years of Newborn Screening for Pompe Disease by CAP Nov 20 2015 News November 18 2005 was a special day It was the day that the first patient identified with Pompe disease by newborn screening NBS was born in Taiwan She was fortunate that she was born in a hospital where the pilot program started only eighteen days before she was born and that she was born after many people worked tirelessly to develop a diagnostic method suitable for NBS Rare Disease Group Urges Ontario to Include Devastating Neuromuscular Condition in Newborn Screening Program by CAP Sep 11 2015 Press Releases TORONTO Sept 11 2015 The Canadian Association of Pompe CAP today submitted a nomination for Pompe disease to be considered for addition in Ontario s newborn screening panel In recognition of Newborn Screening Awareness Month CAP urges Newborn Screening Ontario to expand its program to include this rare neuromuscular condition Without early diagnosis and treatment infants with this rare genetic illness are not likely to survive past the age of one 2015 CAP Conference and AGM Save the Date by CAP Mar

    Original URL path: http://www.pompecanada.com/blog/category/news/ (2016-02-13)
    Open archived version from archive

  • Press Releases Archives - Canadian Association of Pompe
    that its investigational new drug IND application submitted to U S Food and Drug Administration FDA is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1 2 study of ATB200 in patients with Pompe disease Rare Disease Group Urges Ontario to Include Devastating Neuromuscular Condition in Newborn Screening Program by CAP Sep 11 2015 Press Releases TORONTO Sept 11 2015 The Canadian Association of Pompe CAP today submitted a nomination for Pompe disease to be considered for addition in Ontario s newborn screening panel In recognition of Newborn Screening Awareness Month CAP urges Newborn Screening Ontario to expand its program to include this rare neuromuscular condition Without early diagnosis and treatment infants with this rare genetic illness are not likely to survive past the age of one Genzyme Recognizes International Rare Disease Day with Global Education and Fund Raising Events Announces Founding Partnership of Give RARE by CAP Feb 27 2015 Press Releases Genzyme a Sanofi company announced today that in support of International Rare Disease Day the company is hosting several events internationally to help raise awareness of rare diseases as a critical global health issue requiring ongoing research education and new treatments Genzyme also announced that it is a founding partner of Give RARE a rare diseases global giving day Audentes Therapeutics Joins Global Organizations In Celebrating Rare Disease Day 2015 by CAP Feb 26 2015 Press Releases SAN FRANCISCO CA February 26 2015 Audentes Therapeutics Inc a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious rare diseases today announced its support for Rare Disease Day to be held on February 28th 2015 The theme of this year s Rare Disease Day is Living with a Rare Disease In honor of the day Audentes

    Original URL path: http://www.pompecanada.com/blog/category/news/press-releases/ (2016-02-13)
    Open archived version from archive

  • CAP News Archives - Canadian Association of Pompe
    Pompe disease in Provincial territorial Newborn Screening panels CAP on Orphanet by CAP Feb 18 2014 CAP News We re pleased to announce that the Canadian Association of Pompe is now listed on Orphanet Orphanet is the reference portal for information on rare diseases and orphan drugs for all audiences Rare Disease Day 2013 from CAP by CAP Mar 19 2013 CAP News Happy Rare Disease Day everyone That does

    Original URL path: http://www.pompecanada.com/blog/category/cap-news/ (2016-02-13)
    Open archived version from archive

  • 2015 CAP Conference and AGM - Save the Date - Canadian Association of Pompe
    Healthcare Conference Sanofi Genzyme Announces Recipients of Patient Advocacy Leadership PAL Awards Amicus Therapeutics Commences Phase 1 2 Study of Novel ERT for Treatment of Pompe Disease Categories Awareness CAP News Education Events Interviews Living with Pompe News Press Releases Research Studies Tags advocacy awards Awareness CAP chaperone CORD eLearning Emma Rooney ERT Extraordinary Measures Fabry fundraising Gaucher gene therapy getting treated grants guest blogger Int l Pompe Day IPA

    Original URL path: http://www.pompecanada.com/blog/news/2015-cap-conference-and-agm-save-the-date/ (2016-02-13)
    Open archived version from archive

  • Genzyme Recognizes International Rare Disease Day with Global Education and Fund-Raising Events; Announces Founding Partnership of Give RARE - Canadian Association of Pompe
    Genetics divisions of six hospitals and five different patient organizations in Bangkok Belgium Genzyme s Brussels office will offer an educational program to employees on what it means to live with a rare disease and will support the global theme of Living with a Rare Disease Day by Day Hand in Hand South Korea Genzyme in conjunction with the non profit organization Good People International will collaborate with the Seoul Metropolitan Government and Korean Society of Medical Genetics for the 6 Minute Walk campaign Germany Employees will track their steps for one week in a pedometer challenge that will underscore their Rare Disease Day theme Keep Moving for Rare Diseases Day by Day Step by Step Colleagues in Switzerland and Austria are also participating in the pedometer challenge Japan Employees will participate in Genzyme s signature relay race and will make a financial contribution to a local patient organization for each participating runner France Genzyme has formed a partnership with the patient association Alliance Maladies Rares in a program that helps explain rare diseases to children through the local newspaper Mon Petit Quotidien Vietnam On February 26 Genzyme supported National Pediatrics Hospital s Rare Disease Day event which included speeches from patients doctors and the hospital s senior leadership Australia Rare Disease Day will be marked with an awareness event for Genzyme employees The office will also support the National Plan for Rare Diseases Summit hosted by Rare Voices in Australia Italy On March 1 Genzyme Italy employees will join community members in the Modena Run4Rare relay Funds raised will be used to purchase a special swing in the local playground for children with disabilities Spain Genzyme Spain will support the Spanish Federation for Rare Diseases FEDER event that will focus on raising awareness and education around rare diseases China Genzyme will support a press conference in Shanghai on Rare Disease Day to announce the newly formed Rare Disease Expert Advisory Committee and share achievements and future plans for the Shanghai Rare Disease Fund UK Employees will run a 1 3 5 or 10k distance to show their support for rare disease patients Employees will also participate in a series of policy focused events at parliament sites in Cardiff Edinburgh London and Belfast Malaysia Genzyme in Malaysia will host a Rare Disease Day event for employees on February 27 that will include a presentation from a local physician who will share his experience working with patients with rare diseases About Genzyme a Sanofi Company Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years We accomplish our goals through world class research and with the compassion and commitment of our employees With a focus on rare diseases and multiple sclerosis we are dedicated to making a positive impact on the lives of the patients and families we serve That goal guides and inspires us every day Genzyme s portfolio of transformative therapies which are marketed in countries around

    Original URL path: http://www.pompecanada.com/blog/news/press-releases/genzyme-recognizes-international-rare-disease-day-with-global-education-and-fund-raising-events-announces-founding-partnership-of-give-rare/ (2016-02-13)
    Open archived version from archive

  • Blog - Canadian Association of Pompe
    the plan using its Drugs for Rare Diseases Framework The framework was established to assess drugs using the best available evidence and recommend drugs for funding based on clinical criteria read more Woman with Rare Disease Protests at NB Legislature to get Treatment by CAP Apr 16 2014 News Still struggling to receive the same treatment that patients in other provinces receive Annette Sebey of Perth Andover New Brunswick celebrates International Pompe Day in front of the NB Legislature read more Update from Genzyme on neoGAA Pompe clinical development program 11th February 2014 by CAP Mar 3 2014 Research Studies Genzyme is in the early stages of investigating a next generation enzyme replacement therapy neoGAA for Pompe disease It differs from the original ERT whereby carbohydrates are bonded onto the enzyme during the complex manufacturing process of neoGAA These carbohydrates are thought to help the enzyme enter the muscles more efficiently Though it s still very early in the development process and there are many unknowns the hope is that through this more efficient uptake into the muscle cell neoGAA might improve on the effectiveness of the existing therapy read more Rare Disease Day 2014 from CAP by CAP Feb 28 2014 News Happy Rare Disease Day everyone Today raises awareness tolerance and understanding It s amazing how many people are affected by the array of rare diseases out there Collectively we re not so rare after all read more Genzyme Recognizes International Rare Disease Day with Global Awareness Programs and Launch of Fourth Annual Patient Advocacy Leadership PAL Awards by CAP Feb 28 2014 Press Releases CAMBRIDGE Mass BUSINESS WIRE Genzyme a Sanofi company EURONEXT SAN and NYSE SNY today announced its support of International Rare Disease Day with a series of initiatives meant to call attention to rare

    Original URL path: http://www.pompecanada.com/blog/page/2/ (2016-02-13)
    Open archived version from archive

  • Awareness Archives - Canadian Association of Pompe
    2010 Awareness A New Brunswick woman with a rare genetic disease hopes the release of a new Harrison Ford movie on Friday will help draw attention to her plight Extraordinary Measures Official Trailer by CAP Jan 30 2010 Awareness More than a movie by CAP Jan 22 2010 Awareness A new Harrison Ford movie hits close to home for an Alberta teen Extraordinary Measures now in theatres tells the true

    Original URL path: http://www.pompecanada.com/blog/category/awareness/ (2016-02-13)
    Open archived version from archive



  •