NDF Patient Day @UCLA  


Saturday August 29, 2015 from 10:00 AM to 4:00 PM PDT
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UCLA, Grand Horizon Ballroom
330 De Neve Drive
Sunset Village, Covel Commons
Grand Horizon Room, Salon A
Los Angeles, CA 90095

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Lale' Welsh, Executive Director

Neuromuscular Disease Foundation

Event is FREE to patients and their guests and Travel Scholarships have been made available through the generous support of Ultragenyx.  

See registration page for details. 



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Please join NDF for our second GNE Myopathy (HIBM) Patient Advocay Day and Luncheon, this time to be held on the West Coast at UCLA's Grand Horizon Ballroom. 

Speakers and panelists will be GNE Myopathy Experts, Doctors, biologists, scientists, patients, family members  and other GNE advocates.  Discussions will center around the mechanisms of GNE myopathy/HIBM gene therapy, advancements in potential treatments in clinical studies, and hear from guests for their suggestions on how we can support and collaborate from a patient-centric perspective toward our common goal.  

Our final agenda is still in formation, however, here's an overview of what you can expect.  

NDF Patient Advocacy Symposium AGENDA***In formation, subject to change.

  • 9:45-10:15AM Check-in, network

  • 10:20 Welcome & opening Remarks (NDF)

  • 10:30 Jennifer (Patient)

  • 10:40 KEYNOTE: Dr. Nuria Carrillo (NIH)

  • 11:05 11:10 Dr. Alison Skinar, (Ultragenyx)

  • 11:25Q&A

  • 11: 30 Dr. Siavash Kurdestani (UCLA)

  • 11:40Q&A

  • 11:45 Dr. Perry Shieh 

  • 11:55Q&A

  • 12:00 -1:00 PM Panel discussion: (Moderated by Dr. Jennifer Caughlin) Includes Dr. Shieh, Dr. Carmen Bertoni, Dr. Laura Rufibach, Dr. Nuria Carrillo, Dr. Skinar.)

  • Panel Discussion/Interactive audience participation Moderated by Jennifer Caughlin, PhD., and Counselor & NDF, Board member, Carol Gelbard Q&A with scientists, physicians & Advocates.

  • 1:00 luncheon served on the adjoining open-air terrace with campus views.

  • 2:00 Break out sessions: Patients, Salon C (led by Jennifer Caughlin, PhD)

  • 2:15 Family & Caretakers, Salon A (led by Carol Gelbard, in tandem with Physicians/Scientists* TBD).

  • 3:45 PM Closing remarks (Salon A) survey/hand-outs

  • 3:50-4PM Fill out surveys, Adjourn. HARD STOP at 4PM. 


If you have any comments, questions or suggestions about the event, please don't hesitate to contact us directly. Space is limited so please register as soon as possible. 


Lale' J. Welsh

Executive Director, 
Neuromuscular Disease Foundation