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Second-Ever Patients Webinar, A Survey for Patients, A Short Video On GNE Myopathy, Ultragenyx Withdrew Its European Application For Sialic Acid

GNE Myopathy International (GMI) held its first-ever patients webinar in September and it was a great success.  We had patients from all over the globe participating in that webinar. Now we are planning a second patients webinar in collaboration with  the Neuromuscular Disease Foundation (NDF) on December 3. Lale' Welsh, the CEO from the NDF has graciously volunteered to be the presenter.  The discussion is on "Recent Developments in Treatments for GNE Myopathy."  Please refer to the attached flyer on how to connect to this upcoming webinar. 
Webinar Flyer And Direct Link to Connect on Dec. 3rd. 8 a.m. Pacific Time:  https://altvil.zoom.us/j/525434317?pwd=&status=success

Survey For Patients
GNE Myopathy International (GMI) has developed a short survey on various treatment options for GNE Myopathy patients. This survey is intended to collate patients driven information on therapies/treatments with the hope that the entire community might benefit.  All survey responses will remain anonymous to all including the ones hosting the survey. We hope to share the data from this survey at our next webinar.  It takes about 10 minutes to complete this survey. 

Link for Survey: https://docs.google.com/forms/d/e/1FAIpQLSeyI9xIaLN6BVQJHcWUUw2PIHcYRA25IfYcvm6cABlwyZBW8w/viewform?c=0&w=1

The Neuromuscular Disease Foundation has prepared a short and relevantly meaningful video on GNE Myopathy. It gives a brief overview of the earliest historical transmission of the GNE gene, its diaspora, its challenges, and the current therapies for GNE Myopathy.  I feel very humbled to be featured in a small part of this video.

Bracing For Life With GNE Myopathy Video:


Ultragenyx's Withdrawal of Marketing Application of Sialic Acid in the European Union

On November 11, Ultragenyx announced the withdrawal of the Marketing Authorization Application (MAA) for Aceneuramic Acid Prolonged Release (Ace-ER or Sialic Acid) in the European Union. Ace-ER (Sialic Acid) is used  in ongoing trials for patients with GNE Myopathy.

Data based on the Phase 2 study of Sialic Acid was presented to the European Committee for Medical Products for Human Use (CHMP).  This Committee agreed that the Phase 2 study was encouraging but "did not provide sufficient amount of evidence to support approval at this time."

Meanwhile, Ultragenyx aims to collect more information based on the efficacy of Ace-ER on patients who are currently enrolled in the Phase 3 trial. Ultragenyx's CEO Dr. Kakis, stated that "Our Phase 3 study is on track and is designed to confirm the encouraging results seen in Phase 2 study." Dr. Kakis further emphasized that Ultragenyx is "committed to bringing this potential therapy," to GNE Myopathy patients.
Here is a link for the complete article:

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