News Archives

Needle Core Muscle Biopsy

Apr 3, 2012 | WEBINARS

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March 2012 Community Update From Genzyme Regarding 160L Myozyme Supply

Mar 20, 2012 | NEWS

March 19, 2012 Community Update for Pompe Families in the United States who Utilize Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) Produced at the 160 L Manufacturing Scale...

Needle Core Muscle Biopsy

Mar 7, 2012 | PAST WEBINARS

Webinar Details Title: Needle Core Muscle Biopsy Date: Wednesday, March 7, 2012 Speaker:Mark Tarnopolsky, MD, PhD, FRCP (C) Webinar Overview:Traditionally most Neuromuscular Clinics have used an open muscle biopsy for pathological diagnosis. Many Neuromuscular...

2012 PCMA Pull for Pompe Fundraiser

Feb 26, 2012 | NEWS

PCMA of Texas invites you to enjoy a day of family fun benefiting Acid Maltase Deficiency, also known as Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. This event is designed for all ages and...

NMR Imaging And Spectroscopic Techniques For Diagnosis, Follow Up And Evaluation Of Pompe Disease

Jan 24, 2012 | PAST WEBINARS

Webinar Details Title: NMR Imaging (MRI) and spectroscopic (NMRS) techniques for diagnosis, follow up and evaluation of Pompe disease Date: Tuesday, January 24, 2012 Speakers:Robert-Yves Carlier, MDPascal Laforêt, MDDavid Orlikowski, MD, PhDClaire Wary, PhD Webinar...

Announcement Regarding 160 liter Myozyme Supply

Jan 17, 2012 | NEWS

As some of you are probably aware, Genzyme has announced that, due to production and supply constraints, starting March 30, 2012 the 160 liter MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More (which is...

Conference Report Available for 2011 AMDA/IPA Patient and Scientific Conference

Jan 15, 2012 | CONFERENCES

2020AOn October 7-9, 2011, 150 Pompe patients and their families, scientists, doctors, and industry representatives from around the world gathered in San Antonio, Texas for the 2011 AMDA/IPA Pompe Patient and Scientific Conference. Over the course of this weekend,...

BioMarin Receives Orphan Drug Designation from FDA for Pompe Disease

Aug 30, 2011 | NEWS

Excerpt from August 30, 2010 press release . . . BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and...

Support for Proposed Legislation in California Regarding Prior Authorization

Aug 30, 2011 | NEWS

Hi All, The AMDA has been working with Pfizer on supporting some proposed legislation in California that is intended to streamline the process to get prior authorization of medication. The proposed Bill is SB 866. This Bill could be taken up by the House and Senate...

News August 2010 Pompe Program Update from Genzyme

Aug 10, 2011 | NEWS

Genzyme would like to provide an update to the US Pompe community regarding the transition to commercial LumizymeTM (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) from the ATAP...

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