The Heier Laboratory

Researching next generation treatments and biomarkers for pediatric diseases

[The Heier Laboratory @TheHeierLab]

We're hiring! We are excited to be in our new home @VCU in Richmond, VA and are actively recruiting lab members at all levels! If you're interested in basic or translational science in dystrophic muscle 💪 or heart ❤️, contact us!

[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]

Great First Person author interview with Trinitee! Thank you for the great journal experience, and for highlighting promising young scientists @DMM_Journal

https://t.co/ezU7je8xHn

[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

[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

[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

[The Heier Laboratory @TheHeierLab]

Our new paper is out!
Vamorolone improves disease and increases dystrophin in our Becker muscular dystrophy model.
Now in clinical trials (NCT05166109)
#MuscularDystrophy #Becker #dystrophin
@FiorilloLab @MDAorg @ParentProjectMD @nih_nhlbi @iScience_CP
https://t.co/niTwAh3zyy

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

Pleased to announce that the PDF file of our article on DG9-conjugated #morpholino and rescue in #SMA mice is now available @JCI_insight. This promising delivery strategy reached the CNS via an SC administration. https://t.co/BFI16K6DDd @UAlberta_FoMD supported by @WCHRIUofA

[Mattia Quattrocelli @mattiaquattro]

Happy to share out latest methods paper on: Comprehensive Analyses of Muscle Function, Lean and Muscle Mass, and Myofiber Typing in Mice https://t.co/h5rgeEHB7d

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

Thrilled to share our latest findings on the potential of DG9-conjugated morpholino as a treatment for spinal muscular atrophy! Our study shows significant improvements in survival, SMN expression, and muscle function in a mouse model. @UAlberta_FoMD https://t.co/gAkfsar3Kr

[The Heier Laboratory @TheHeierLab]

Pop Quiz!

[Children's National Hospital 🏥 @ChildrensNatl]

Test your muscular dystrophy knowledge: Which muscles are sick and how do you know? #MedEd #MedTwitter

[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

[Alyson Fiorillo, PhD @AlysonAFiorillo]

I am so excited to share our newly published work on generating and characterizing the first mouse model of #Beckermusculardystrophy @MacGyver0282 @TheHeierLab @nikkimmccormack @EdgewiseTx @FiorilloLab #duchennemusculardystrophy #dystrophin https://t.co/AJ1ZNNpcN4

[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

[W. David Merryman @merrymanlab]

If @NIHFunding raised the modular budget cap, what should it be raised to?

[Mattia Quattrocelli @mattiaquattro]

Only two weeks (and two days) to our first Circadian Biology Workshop at Cincinnati Children's - University of Cincinnati. Looking forward to new data, exciting new ideas and new collaborations in the making @CincyChildrens @CincyKidsOAACD @uofcincy

[GW Integrated Biomedical Sciences @GW_IBS]

A huge round of applause 👏🏽 for the newly-minted Dr. Kelly Murphy, who has defended her dissertation investigating antisense oligonucleotide (AON) therapy for facioscapulohumeral #MuscularDystrophy (FSHD) in the Chen lab! 🎉💪🏽 #GWIBSFamily #DissertationProps #WomenInSTEM #PhDLife

[Mattia Quattrocelli @mattiaquattro]

Our lab is hiring postdocs to study muscle and heart function and disease through the perspectives of circadian, metabolic and epigenetic mechanisms. Our lab is committed to diversity, equity and inclusion and supports all career aspirations @CCHMCPostdoc