The Heier Laboratory (@TheHeierLab) header
The Heier Laboratory (@TheHeierLab) profile picture

The Heier Laboratory

@TheHeierLab

Researching next generation treatments and biomarkers for pediatric diseases

 Washington, DC
heierlab.org/
Joined September 2020
  • Tweets40
  • Following362
  • Followers145
Happy to share our latest article: Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping: Molecular Therapy - Nucleic Acids cell.com/molecular-therapy-f…. Congrats to first author and talented post-doc @nikkimmccormack! #DMD #microRNA (1/3)
Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping

Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping

Fiorillo and colleagues found elevated miR-146a-5p in DMD and BMD muscles. They show miR-146a-5p is induced by inflammation and inhibits dystrophin translation. Using mdx52 mice with genetic deletion...

cell.com
1
2
3
226
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: mdaquest.org/agamree-vamorol…
Image from Muscular Dystrophy Association (@MDAorg)
Image from Muscular Dystrophy Association (@MDAorg)
Image from Muscular Dystrophy Association (@MDAorg)
Image from Muscular Dystrophy Association (@MDAorg)
2
3
1
12
1,683
The Heier Laboratory retweeted
CureDuchenne (@CureDuchenne) profile picture
CureDuchenne@CureDuchenne26 Oct 2023
“CureDuchenne is proud to have contributed to the early development of vamorolone at ReveraGen." cureduchenne.org/general/fda…
Image from CureDuchenne (@CureDuchenne)
3
13
776
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. worldduchenne.org/news/fda-a…
FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy • World Duchenne

FDA Approves Vamorolone as a Treatment for Duchenne Muscular Dystrophy • World Duchenne

The U.S. Food and Drug Administration (FDA) has granted its approval for Vamorolone, a new treatment for Duchenne Muscular Dystrophy (DMD).

worldduchenne.org
17
2
38
3,327
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. ⬇️ innovationdistrict.childrens…
New Becker muscular dystrophy drug on the horizon

New Becker muscular dystrophy drug on the horizon

(2 min. read)

innovationdistrict.childrensnational.org
11
40
2,831
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. insight.jci.org/articles/vie… @UAlberta_FoMD supported by @WCHRIUofA
Image from Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair (@ToshifumiYokota)
1
3
15
1,253
The Heier Laboratory retweeted
Mattia Quattrocelli (@mattiaquattro) profile picture
Mattia Quattrocelli@mattiaquattro20 Feb 2023
Happy to share out latest methods paper on: Comprehensive Analyses of Muscle Function, Lean and Muscle Mass, and Myofiber Typing in Mice bio-protocol.org/en/bpdetail…
2
9
1
44
6,040
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 insight.jci.org/articles/vie…
Image from Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair (@ToshifumiYokota)
Image from Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair (@ToshifumiYokota)
Image from Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair (@ToshifumiYokota)
Image from Prof. Toshi Yokota PhD FCAHS @MD_Canada Res Chair (@ToshifumiYokota)
5
9
1
34
6,794
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. spr.ly/60173pSMX

Becker Muscular Dystrophy Center for Genetic Medicine Research

6
19
1,588
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 doi.org/10.1002/jcsm.13171
The X‐linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of...

The X‐linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of...

Background Becker muscular dystrophy (BMD) is a genetic neuromuscular disease of growing importance caused by in-frame, partial loss-of-function mutations in the dystrophin (DMD) gene. BMD presents...

onlinelibrary.wiley.com
4
9
1
31
3,657
The Heier Laboratory retweeted
W. David Merryman (@merrymanlab) profile picture
W. David Merryman@merrymanlab3 Nov 2022
If @NIHFunding raised the modular budget cap, what should it be raised to?
5%$300k
30%$350k
55%$400k
10%Other
80 votes • Final results
3
1
1
The Heier Laboratory retweeted
Mattia Quattrocelli (@mattiaquattro) profile picture
Mattia Quattrocelli@mattiaquattro17 Aug 2022
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
Image from Mattia Quattrocelli (@mattiaquattro)
2
6
35
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
GW IBS PhD defense Kelly Murphy with members of her committee and guests

ALT GW IBS PhD defense Kelly Murphy with members of her committee and guests

2
4
17
The Heier Laboratory retweeted
Mattia Quattrocelli (@mattiaquattro) profile picture
Mattia Quattrocelli@mattiaquattro12 Jul 2022
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
Image from Mattia Quattrocelli (@mattiaquattro)
37
1
61
#5 in the nation. #1 in the nation for newborn care, 6 years in a row. #1 in the mid-Atlantic for neuro care, 7 years in a row. Learn more about what this year’s @usnews rankings mean for families everywhere: spr.ly/6016zjDC2
Image from Children's National Hospital 🏥 (@ChildrensNatl)
30
24
15
64
Congratulations to Drs. Saifullah, Motohashi, Tsukahara, and Aoki for their review article published on the Development of Therapeutic RNA Manipulation for Muscular Dystrophy in our Research Topic frontiersin.org/article/10.3…
2
1
9
Hey #ScienceTwitter —I need your hive mind. Are there any groups our companies that synthesize custom circular RNAs? I’m looking to try to inhibit a group of microRNAs in Duchenne model mice in vivo using this circular RNAs. #microRNAs #Duchenne
2
2
2
The Heier Laboratory retweeted
Joseph Allen (@j_g_allen) profile picture
Joseph Allen
@j_g_allen10 Jan 2022
Childrens Hosp of Phil, Policy Lab —>”closing schools will do nothing to change the number of people showing up at the ER” —>”The primary intervention to protect our families is vaccination, and boosters, not disrupting schools. It’s time to move forward” washingtonpost.com/education…
Perspective | Time to reset coronavirus school policies. Here’s a road map.

Perspective | Time to reset coronavirus school policies. Here’s a road map.

A set of practical recommendations by experts at Children's Hospital of Philadelphia — which may seem counterintuitive to some — is aimed at keeping as many kids in school as possible.

washingtonpost.com
34
61
12
274
Happy first day of classes! We are starting off the new year with a new cover photo, courtesy of our #GWIBS PhD student Lana Jagannathan, who won an Art of Science prize for this image from her rotation in @TheHeierLab!
Image from GW Integrated Biomedical Sciences (@GW_IBS)
1
6
The Heier Laboratory retweeted
Jyoti K Jaiswal (@jyoti_k_jaiswal) profile picture
Jyoti K Jaiswal@jyoti_k_jaiswal4 Jan 2022
Culmination of our studies over the past decade has led to a mechanistic and preclinical study out in @jclinicalinvest. We developed a non-muscle targeted #LGMD 2B #genetherapy that safely improved muscle fibre repair and muscle health jci.org/articles/view/141295…
Image from Jyoti K Jaiswal (@jyoti_k_jaiswal)
16
19
4
93
Load more