Christopher Heier

Using science to solve problems and help people.

[Alyson Fiorillo, PhD @AlysonAFiorillo]

Happy to share our latest article: Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping: Molecular Therapy - Nucleic Acids https://t.co/I9S1nAHH4B. Congrats to first author and talented post-doc @nikkimmccormack! #DMD #microRNA (1/3)

[The Heier Laboratory @TheHeierLab]

Our new paper is published! Learning more about how steroids work in #Duchenne. Congrats to Trinitee on her first 1st author paper! @DMM_Journal has a nice interview with her, link to follow.
@MDAorg @ParentProjectMD @FiorilloLab @MYOTWlTTER #DMD

https://t.co/LQaffG2Kod

[Muscular Dystrophy Association @MDAorg]

The recent @fda approval of the #MDA-Supported Drug AGAMREE® (vamorolone) from @catalystforrare is a beacon of hope for people living with #Duchenne #MuscularDystrophy (#DMD), a rare but serious degenerative muscle disease. Learn more in #Quest: https://t.co/xSG8r61YUw

[CureDuchenne @CureDuchenne]

“CureDuchenne is proud to have contributed to the early development of vamorolone at ReveraGen."
https://t.co/5Qe3qa1GYb

[Muscular Dystrophy Association @MDAorg]

#MDA celebrates the @US_FDA approval of AGAMREE® (vamorolone) to treat children and adolescents living with #Duchenne #MuscularDystrophy (#DMD). Learn more about this novel therapy, supported in part by MDA Venture Philanthropy: https://t.co/c9DfdgqPVW

[World Duchenne Organization @worldduchenne]

Today, the U.S. Food and Drug Administration (FDA) has granted its approval for Vamorolone, a new treatment for Duchenne Muscular Dystrophy (DMD) aged 2 years and older.
https://t.co/z1l0vy0NRk

[World Duchenne Organization @worldduchenne]

EMA's CHMP issued a positive opinion in favor of the approval of AGAMREE® (vamorolone) for DMD patients aged 4 years and up.

🤝 The Duchenne community's contributions as funders, research participants, and our support as @Vision_DMD partner were pivotal.

https://t.co/VUF5A4iaJT

[Angus Lindsay @AJCLindsay]

PhD Opportunity #Myotwitter

If you are interested in the mechanisms regulating skeletal muscle adaptability then please apply to join my new lab @UCNZ, Christchurch, New Zealand!

https://t.co/NGVT1lB6m3

Scholarships are available for stipend + tuition fees.

Please RT

[Children's National Hospital 🏥 @ChildrensNatl]

Positive news for boys with #Becker #MuscularDystrophy: Vamorolone improves disease progression with fewer side effects than current treatments. Read more from @TheHeierLab, @AlysonAFiorillo & the Center for Genetic Medicine Research. ⬇️ https://t.co/W17hjommbK

[Christopher Heier @MacGyver0282]

Our new paper is out! Vamorolone improves disease and increases dystrophin in our Becker muscular dystrophy model. Now in clinical trials (NCT05166109) #bmd #Becker #dystrophin #myotwitter
@FiorilloLab @TheHeierLab @MYOTWlTTER

https://t.co/AP5RJyLzoR

[Christopher Heier @MacGyver0282]

@CBKReport Greg Ostertag, when he ran over his own foot with a van

[Matthias Lambert, PhD @MatthiasLambert]

Great time at #MDAconference in Dallas, Texas, sharing my recent work on TPM3-related myopathy. 🧑‍🔬🦓💪
#RareDiseases #Muscle #Science #Myotwitter
@BostonChildrens @harvardmed @MDAorg

[Monkol Lek @theFourier2k]

Great to see @MatthiasLambert @AlysonAFiorillo @HogarthMarshall and other next generation muscle disease research leaders feature in Genetic mechanisms session at #MDA2023 @MDAorg

[Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair @ToshifumiYokota]

It was a pleasure working with co-editors @RikaMaruyama_E, @AlysonAFiorillo, @MacGyver0282, and Dongsheng Duan. Looking forward to more breakthroughs in the field! #ScienceAdvances #NeuroMuscularDiseases

[Christopher Heier @MacGyver0282]

@TheFFBallers 🫴

[Monkol Lek @theFourier2k]

Attending @GordonConf Seminar as invited plenary speaker and extremely disappointed that Handicap ppl are STILL not considered eligible for the Carl Storm travel fellowship even after the big fight I had in 2018. I guess I don’t quite understand what the word minority means 🤷🤷

[Christopher Heier @MacGyver0282]

@LateRoundQB

[The Heier Laboratory @TheHeierLab]

1st mouse model of Becker muscular dystrophy!! Excited our bmx paper came out this week, check it out here:
#Beckermusculardystrophy @AlysonAFiorillo @FiorilloLab @nikkimmccormack @EdgewiseTx @MDAorg @CureBMD @MDUK_News @ParentProjectMD #dystrophin https://t.co/eOURmuc28o

[Children's National Hospital 🏥 @ChildrensNatl]

Investigators at the Center for Genetic Medicine Research @AlysonAFiorillo & @MacGyver0282 created a model to test new treatments for Becker muscular dystrophy, providing hope to the growing number of patients with this debilitating neuromuscular disease. https://t.co/KFq37WqiFx