Joshua Frase Fnd.
@thejoshuacause
Forging a path for #myotubularmyopathy, a rare & often fatal muscle disorder in children. #changestartswithone #RareDisease #GeneTherapy PleaseDonate.org
ID: 342854413
http://www.joshuafrase.org 26-07-2011 17:49:47
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Thank you Lisa Nickerson for your years of love and support! #myotubularmyopathy #Raredisease
Tamar Rubinstein Yaffa Rubinstein Peter Robinson Melissa Haendel HPO Monarch Initiative National Library of Medicine NCATS Rare Disease Day CRMO SJIA Foundation CRMO Foundation Julie McMurry, MPH We salute our rare disease patients and their families who courageously advocate for change through Open Science. Honored to collaborate on this paper. #RareDiseaseDay2021
An important interview with two influential Grandfathers in the @genetherapy space for Alex O'Meara AT132 for XLMTM Astellas Gene Therapies .. My takeaway - we cannot be silos of data when precious lives are at stake!! Asklepios BioPharmaceutical, Inc. (AskBio) Passage Bio Human Gene Therapy NIH