NDF Spring Newsletter 2020
During these uncertain times, we want to reach out, wish everyone our best and assure you that we at  NDF continue to work tirelessly on behalf of patients  worldwide in our efforts to fulfill our mission .

On the heels of NDF's most successful year to date, we are pleased to present our ANNUAL REPORT for 2019, which details our accomplishments and offers a complete list of our generous donors.

With the funds raised to date, we are able to continue with remaining preclinical studies required by the FDA towards gene therapy in a way that is both efficacious and safe for our patients. In spite of fears and delays surrounding COVID-19 and restrictions placed on our researchers,  NDF remains steadfast in its commitment to see these studies through in as timely a manner as is possible  given our new limitations.

All of us at NDF wish each of you the very best during what we know may be a difficult time for you and your family.  Please feel free to reach out to us if we can be of help in any way. And, for those of you celebrating, we wish you a happy and healthy Nowruz, or Spring Equinox, and with new beginnings.
Where is NDF with Gene Therapy?
While very exciting and hopeful, gene therapy is a new, complicated and lengthy process that requires extreme clarity on the disease itself, and on the type and amount of dosage to be both safe and effective.

For more information on Gene Therapy and what that is visit our site
To understand where we are in this process, please watch this:
NDF is pleased to announce its funding for the following list of r esearch projects in 2020*:  

  1. Dr. Paul Martin, The Research Institute at Nationwide Children’s Hospital, Columbus
  2. Dr. Monkol Lek, Lek Lab -Yale University, New Haven
  3. Dr. Kelly Crowe, Mount St. Joseph University, Cincinnati
  4. Dr. Noah Weisleder, The Ohio State University, Columbus
  5. Dr. Wakako Yoskioka, National Institute of Neuroscience, Tokyo
  6. A second year of a 2-year grant for Dr. Stella Rosenbaum, Hadassah Medical Center, Jerusalem
  7. Continued work on WGS with Dr. Madhuri Hegde, Perkin Elmer, Yale University
  8. Dr. Marjian Huizing & Dr. William A Gahl, NIH, Washington, D.C.

NDF is proud to support NIH with gift funds in support of ManNAc, now is its final clinical phase. To learn more about the ManNAc trials, click here.

We are very grateful to our extraordinary Scientific Advisory Committee and Scientific Proposal Review Team who are essential partners in our continued progress .

*as of date of publication, final confirmations of some contracts are pending due to temporary, COVID-19 limitations of some recipients. Public announcements with more details will be released upon execution of agreements ASAP.
Please click here for a statement about the postponement of our Symposium.  We look forward to welcoming patients, family members, scientists and industry professionals in Boston, when it is deemed safe to do so, for what we know will be a meaningful and productive event. 
NDF Talks
NDF is excited to introduce our new series of podcasts in 2020
Each episode will feature a GNE Myopathy community member’s personal experience with GNEM.
Participants include patients, scientists, caregivers and family members, all sharing the triumphs and sometimes tribulations of life with this rare disease in order to provide potentially helpful and supportive information for anyone whose life is touched by GNEM.

We'd love to share your story. Please e-mail events@curehibm.org to schedule an interview. We'll even let you see the questions first 😄

A message about next phase of ManNAc trial:

What we have been told is that they are still moving forward with the study as they are able. Most hospitals and large academic centers like those participating in this study as clinic sites have restrictions at the moment at ability to enroll new research participants, but they are monitoring the situation closely and still plan to move forward with the trial when able.
NDF will be holding a Special Edition Patient HUDDLE this month on March 29th at 9AM PT facilitated by NDF community member and past board member, psychotherapist Carol Gelbard, L.C.S.W.

This will be a VIDEO meeting so please make sure to have your cameras on.

To register and get instructions
Click HERE
Look at what we accomplished
together in 2019!
Just for Fun
Here's a fun video of Rana Mansour's awesome performance at our last gala for you to enjoy in celebration of Nowruz, or the spring equinox that signifies new beginnings.