Wed, Nov 09, 2016
November Happenings
Category: General
There has been so much going on the past few months ~ here is a recap on the happenings... 
Taylor Hicks and I chilling at the 2016 Global Genes Summit ~ Great Guy!
Wed, Jun 08, 2016
European Adventures-Tour for a Cure!
Category: General
In May, I had the trip a lifetime! I was given the opportunity to visit our European MTM families, and all the pieces to make this trip possible fell into place.
Fri, Apr 22, 2016
The Pursuit to Cure MTM Born our of a Mother's Vision
Category: General
Dear JFF Friends and Families,
20 years and counting...
We want to share with you another milestone met by our MTM community.
Awareness and education are key components to the success of any orphan drug program, and I'd like to share that Alison has recently been published online by the longest running gene therapy magazine, Human Gene Therapy. The attached electronic file link . We are told that the article will be in print in the May issue of Human Gene Therapy.
This article is for the entire community, so please enjoy the read, and know that each in our MTM/CNM community plays an integral part in the success of this journey.
Always for the children.
Best regards,
Paul Frase
Thu, Apr 14, 2016
2nd Generation of The International Family Registry for Centronuclear and Myotubular Myopathies
Category: General
The 2nd Generation of The International Family Registry for Centronuclear and Myotubular Myopathies is now LIVE!
We are excited to share a new movement in the rare disease space! The International Family Registry for Centronuclear and Myotubular Myopathies is releasing its next generation. We are now collaborating with the NIH/NCATS GRDR Program, the Global Rare Disease Registry, Alan Beggs PhD and Casie Genetti, MS, CGC Research Coordinator/Genetic Counselor for Beggs' lab.
Through this new collaboration, our de-identified data will be integrated into the GRDR, allowing investigators to better query patient data and accelerate research across many rare diseases, with the goal of eventually developing novel diagnostics and therapeutics.
We are asking for your help! If you have previously participated in the International Family Registry for Centronuclear and Myotubular Myopathies or have not yet had the opportunity to register with us, please follow the link below to learn more about our new registry and consider taking a few moments to register yourself or your loved one with centronuclear and myotubular myopathies!
**Registry link**
If you have any questions, please do not hesitate to contact connect@joshuafrase.org
Alone we are rare, together we are strong!
Currently the registry is in five languages, English, French, Italian, Spanish and German. Please note, the first page is in english then translates to the language of your choice. If you have a request for a different translation, please don't hesitate to make that request as we have the ability to translate into 90 languages.
Always for the Children!
The Joshua Frase Foundation
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