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  • politics Archives - Canadian Association of Pompe
    some 2 7 million Canadians are affected by 1 in 6 000 rare disorders such as cystic fibrosis sickle cell disease and thalassemia Most rare disorders are difficult to diagnose and are chronic degenerative progressive and life threatening 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

    Original URL path: http://www.pompecanada.com/blog/tags/politics/ (2016-02-13)
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  • pregnancy Archives - Canadian Association of Pompe
    Research Studies An article published on PubMed gov Dec 2011 describes a 40 year old woman with Pompe Disease who successfully carried a healthy baby to term 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

    Original URL path: http://www.pompecanada.com/blog/tags/pregnancy/ (2016-02-13)
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  • Rare Disease Archives - Canadian Association of Pompe
    topic of rare disease in Canada how it affects so many of us and what changes are forthcoming S O S Help Make a Difference in Ian s Life by CAP Nov 12 2007 Living with Pompe 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

    Original URL path: http://www.pompecanada.com/blog/tags/rare-disease/ (2016-02-13)
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  • Rare Disease Day Archives - Canadian Association of Pompe
    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 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 diseases as an important public health issue and to improve rare disease education research and treatment Amicus Therapeutics Inc FOLD to Ring The NASDAQ Stock Market Closing Bell by CAP Feb 27 2014 Press Releases Amicus Therapeutics Inc a biopharmaceutical company at the forefront of therapies for rare and orphan diseases will visit the NASDAQ MarketSite in Times Square in celebration of Rare Disease Day 2014 Rare Disease Day 2014 by CAP Feb 13 2014 Events 28 February 2014 marks the seventh international Rare Disease Day coordinated by EURORDIS On and around this day hundreds of patient organisations from more than 70 countries and regions worldwide are planning awareness raising activities around the slogan Join Together for Better Care Rare Disease Day 2013 from CAP by CAP Mar 19 2013 CAP News Happy Rare Disease Day everyone That does seem like an odd thing to say but we celebrate the things that define our lives and for those of us that have a rare disease it certainly plays a significant part in it Genzyme Recognizes International Rare Disease Day with Second Annual Patient Advocacy Grant Program by CAP Feb 28 2012 Press Releases CAMBRIDGE Mass BUSINESS WIRE Genzyme a Sanofi company

    Original URL path: http://www.pompecanada.com/blog/tags/rare-disease-day/ (2016-02-13)
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  • trials Archives - Canadian Association of Pompe
    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 BioMarin to Advance BMN 701 for Pompe Disease to Next Phase of Development by CAP Mar 19 2013 Press Releases SAN RAFAEL Calif March 19 2013 GLOBE NEWSWIRE BioMarin Pharmaceutical Inc Nasdaq BMRN announced today results from POM 001 the Phase 1 2 trial for BMN 701 a fusion protein of insulin like growth factor 2 and acid alpha glucosidase IGF2 GAA for the treatment of late onset Pompe disease First gene therapy trial launched for Fabry disease by CAP Feb 18 2013 Press Releases CALGARY AB Jan 24 2013 Researchers in Calgary have launched the first gene therapy clinical trial in the world for Fabry disease that could ultimately lead to a permanent cure Amicus Therapeutics Announces Positive Preliminary Results From Ongoing Phase 2 Chaperone Enzyme Replacement Therapy ERT Co Administration Study for Pompe Disease by CAP Jun 26 2012 Press Releases CRANBURY N J June 26 2012 GLOBE NEWSWIRE Amicus Therapeutics Nasdaq FOLD today announced positive preliminary results from an ongoing Phase 2 open label drug drug interaction study Study 010 to evaluate the safety and pharmacokinetic PK effects of the pharmacological chaperone AT2220 duvoglustat HCl co administered with enzyme replacement therapy ERT for Pompe disease Amicus Therapeutics Announces FDA Agreement to Commence Phase 2 Study of AT2220 Co administered with Enzyme Replacement Therapy for Pompe Disease by CAP Mar 8 2011 Press Releases CRANBURY N J March 8 2011 PRNewswire Amicus

    Original URL path: http://www.pompecanada.com/blog/tags/trials/ (2016-02-13)
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  • video Archives - Canadian Association of Pompe
    an unannounced spectacular end to the meeting Clip Syndicate Video Pompe Disease by CAP Jan 20 2011 Awareness Our fellow fighter of Pompe Disease Monique Griffin is featured in this video Monique is the author of M E G s Confessional Extraordinary Measures Official Trailer by CAP Jan 30 2010 Awareness Recent Posts Inequities Between Provinces in Treating Rare Diseases Rare Disease Strategy Urgently Needed to Help 3 Million Canadians

    Original URL path: http://www.pompecanada.com/blog/tags/video/ (2016-02-13)
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  • Ten Years of Newborn Screening for Pompe Disease - Canadian Association of Pompe
    she had symptoms she could be monitored When her symptoms appeared at about one year of age she started to receive Myozyme Had she not been identified through NBS she and her family would have embarked on the diagnostic odyssey that most Pompe patients travel They were spared the confusion and pain that s caused by progression of the disease and by simply not knowing what s wrong To be the first at anything takes a lot of work There are many people that made it possible for this patient to be diagnosed be they researchers doctors nurses lab techs administrators etc We would never be able to identify them all here and doing so would distract us from the story We hope everyone that contributed reads this accepts our thanks and knows that they contributed to something important that saves lives Tagged ERT newborn screening Share this Facebook Twitter Google LinkedIn Related 1 Comment Loretta on November 21 2015 at 9 06 am I have a Granddaughter with Pompeii she is now 8months old she was diagnosed at 3 months I love getting all the information Thank you for all you do Reply Leave a Reply to Loretta Cancel reply Your email address will not be published Required fields are marked Comment Name Email Website 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

    Original URL path: http://www.pompecanada.com/blog/news/ten-years-newborn-screening-pompe-disease/?replytocom=34 (2016-02-13)
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  • CAP Supports Newborn Screening for Pompe Disease - Canadian Association of Pompe
    infant onset patients are so severely affected very little discussion was needed to unanimously support screening for such a devastating disease It s so crucial for these patients to receive timely treatment Eliminating the time required for diagnosis can be a life saving choice certainly affecting quality of life We felt there were no significant cons and fully support screening for infant onset Pompe disease Most of our discussion was spent weighing the pros and cons of screening for adult onset Pompe disease Our concerns were false positives the burden on the patient family of knowing the potential burden of treatment before treatment is necessary and the burden of reproductive choice After very careful consideration our reasons for choosing to support screening for late onset Pompe disease were eliminating the diagnostic journey timely and correct treatment with active management throughout and reproductive choice We recognize that current screening would identify both infant onset and late onset versions of Pompe disease Our belief is that the cons we ve discussed are outweighed by the pros for late onset Pompe patients and far outweighed by the benefit to infant onset Pompe patients We hope that our perspective as patients families will be valuable in decisions going forward Please contact me at anytime to discuss these issues further Best Regards Brad Crittenden President Tagged newborn screening CAP NBS Position Letter 2014 File size 194 kB 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

    Original URL path: http://www.pompecanada.com/blog/cap-news/cap-supports-newborn-screening-for-pompe-disease/ (2016-02-13)
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