Joshua Frase Fnd.

Forging a path for #myotubularmyopathy, a rare & often fatal muscle disorder in children. #changestartswithone #RareDisease #GeneTherapy

[Joshua Frase Fnd. @TheJoshuaCause]

Not all #superheroes wear capes! Outstanding positive outcomes in Phase 1/2 in clinical trials. It all began here, 22 years in the works. #humbled #HopeHasNoBounds #GeneTherapy https://t.co/ELCDPxpWc1

[Joshua Frase Fnd. @TheJoshuaCause]

GivingTuesday = GivingHope - Please consider giving on the most philanthropic day of the year.
The Joshua Frase Foundation is approaching its 30th anniversary, and we are more convinced than ever that the mission of saving lives is a journey that has been ordained. We wholeheartedly believe that we have a job to finish, and we will not rest until our HOPE is realized in a therapy or a cure for children afflicted with the deadly disorder of myotubular myopathy (MTM).

https://t.co/JRRHlqHKks

#giving #givingback #GivingTuesdaycampaign #GlobalImpact #savinglivestogether #togetherwearestronger

[Joshua Frase Fnd. @TheJoshuaCause]

@Squawk55 What’s your TikTok

[Joshua Frase Fnd. @TheJoshuaCause]

Happy Birthday Son - Life is a Gift! - Joshua's life was a door to great expectations and a vehicle of hope. Until we see you again son. Much Love https://t.co/59RBUw2BES

[Joshua Frase Fnd. @TheJoshuaCause]

Our Goal; Lasting Change -The journey has been long, and the fight is not over! You have trusted us before: we need your investment in our vision, fortitude and resolve now more then ever. Always for the children. https://t.co/NH6QrwoZT8

[Joshua Frase Fnd. @TheJoshuaCause]

An important interview with two influential Grandfathers in the @genetherapy space for @clinicaltrial AT132 for XLMTM @AstellasGeneTx .. My takeaway - we cannot be silos of data when precious lives are at stake!! @ask_bio @Passage_Bio @HGTJournal @NIH

[Jason Mast @Jasonmmast]

Just under a year ago, the first of three boys died in an Audentes gene therapy trial.

The trial has resumed, but it's still not clear what, precisely, happened or how to stop it from happening again.
https://t.co/eDBA3gpjw4

[Joshua Frase Fnd. @TheJoshuaCause]

@TamarPedsRheum @Yafrubinstein @pnrobins @ontowonka @hp_ontology @MonarchInit @NLM_NIH @ncats_nih_gov @rarediseaseday @Fight_CRMO @sjiafoundation @CRMOFoundation @figgyjam We salute our rare disease patients and their families who courageously advocate for change through Open Science. Honored to collaborate on this paper. #RareDiseaseDay2021

[Joshua Frase Fnd. @TheJoshuaCause]

@TamarPedsRheum @Yafrubinstein @pnrobins @ontowonka @hp_ontology @MonarchInit @NLM_NIH @ncats_nih_gov @rarediseaseday @Fight_CRMO @sjiafoundation @CRMOFoundation @figgyjam We salute our rare disease patients and their families who courageously advocate for change through Open Science. Honored to collaborate on this paper. #RareDiseaseDay2021

[Joshua Frase Fnd. @TheJoshuaCause]

Season of Blessings - https://t.co/LQ6kNxPVYm

[Joshua Frase Fnd. @TheJoshuaCause]

The Fruit of Generosity! As we approach the most philanthropic day of the year, Giving Tuesday, December 1st, we wanted to share a few of our accomplishments we achieved in 2020, against all odds *|https://t.co/BtgLmuUXmq #GivingTuesday

[Joshua Frase Fnd. @TheJoshuaCause]

JFF's 2020 Mission ~ The Fight is Not Over! Now we are at a crossroad, a point of inflection for the future of gene therapy, for MTM and countless other indications. We need your support! #GeneTherapy #clinicaltrials #Children
https://t.co/u85tFptWtq via @YouTube

[Joshua Frase Fnd. @TheJoshuaCause]

A story of perseverance and teamwork ! It was an honor and a privilege to work with the Tanner Pharma Group and ZNM-Zusammen Stark, to allow this family in Equator the ability to bring Wilson home and spare him the life in an institution. https://t.co/I5tjwVUdf2 |*

[Joshua Frase Fnd. @TheJoshuaCause]

@dynacure We applaud Team Dynacure for the advancements being made in the field of neuromuscular disorders!!

[Joshua Frase Fnd. @TheJoshuaCause]

A Tale of Two Clinical Trials: Gene Therapy for a Rare Disease and a Vaccine for COVID-19 - DNA Science https://t.co/NtIm3S3T85 #Genetherapy #Myotubularmyopathy #Audentes #Clinicaltrial #COVID19

[Joshua Frase Fnd. @TheJoshuaCause]

Alison Frase: Mother on a Mission ~ Live with Lisa https://t.co/cxBgVAIIsK via @YouTube. A brief overview on how JFF’s efforts brought an ultra rare disorder to #ClinicalTrials #GeneTherapy #Audentes #PatientAdvocacy #MiracleDog

[Joshua Frase Fnd. @TheJoshuaCause]

Thank you @LisaNickerson for your years of love and support! #myotubularmyopathy #Raredisease

[Lisa Nickerson @LisaNickerson]

I’m so excited - Alison’s interview is already one of the most viewed #LivewithLisa shows! Click through and be inspired by Alison’s mission to find a cure for the #neuromuscular disease #myotubularmyopathy https://t.co/2vU7qgA8oX via @YouTube @AliRockettFrase @TheJoshuaCause

[Joshua Frase Fnd. @TheJoshuaCause]

Please consider this meaningful initiative for Wilson! This child is worth the fight

[Holger Fischer @holfischer]

Please help us, that little Wilson with #CentronuclearMyopathy can be at home with his family. in #Ecuador. @ZNM_Stark is fundraising and informing here: https://t.co/kaFaITCNzM
#SiSePuede #JuntosSomosfuertes #RareDisease

[Joshua Frase Fnd. @TheJoshuaCause]

Amid COVID 19 Crisis, Joshua Frase Foundation Leads Search Spanning Two Continents to Deliver Ventilator to Ailing Child in Ecuador - https://t.co/VdJnUO9bsl

[Joshua Frase Fnd. @TheJoshuaCause]

We live in exciting times! Congratulations Team Dynacure #myotubularmyopathy #CNM #MTM

[Joshua Frase Fnd. @TheJoshuaCause]

The Essence of Life https://t.co/oVmc0yEblG via @YouTube #GlobalRareDiseaseDay2020 Today we honor this community ~ Know we see you, we hear you, we serve you, because we are you! #MyotubularMyopathy #CNM #MTM #ChangeStartsWithOne #Community

[Joshua Frase Fnd. @TheJoshuaCause]

Advancements being made for our CNM/ MTM community. Well done! #myotubularmyopathy #incidence #prevalence