FiorilloLab (@FiorilloLab) profile picture

FiorilloLab

@FiorilloLab

Principal Investigator at Children's National Medical Center. Our lab focuses on dysregulated miRNAs in DMD and other muscle disorders.

Joined July 2021
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  • Following103
  • Followers42
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 cell.com/iscience/fulltext/S…
Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice

Pharmacology; Biological sciences; Neuroscience

cell.com
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Test your muscular dystrophy knowledge: Which muscles are sick and how do you know? #MedEd #MedTwitter
Image from Children's National Hospital 🏥 (@ChildrensNatl)
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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
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Hey #ScienceTwitter; is there a circRNA array/sequencing company/platform that you recommend? And are there any companies out there that synthesize custom cirRNAs?
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FiorilloLab retweeted
Joy Wu, MD, PhD (@JoyYWu) profile picture
Joy Wu, MD, PhD@JoyYWu18 Feb 2022
With our publication in @NatureMedicine on pandemic-related barriers to the success of women in research, I’d like to shine a spotlight on gender equity for women physician-scientists, who face cumulative barriers imposed in medicine AND science rdcu.be/cHd4a 1/n
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Harry's review article "Casimersen for Duchenne muscular dystrophy" is featured in the latest issue of #DrugsofToday @clarivate, summarizing the preclinical and clinical data currently available for casimersen, emphasizing pharmacokinetics and safety. @WCHRIUofA @UAlberta_FoMD
Insira (@Insira_Global) profile pictureInsira@Insira_Global28 Jan 2022
#DrugsofToday >> In the latest issue, @Clarivate experts take a look at #Casimersen, a phosphorodiamidate morpholino oligomer recently approved for the treatment of #DuchenneMuscularDystrophy. Learn more: bddy.me/3o8Xd9w #drugdevelopment #lifesciences #DMD
Image from Insira (@Insira_Global)
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Interesting new @Nature paper. MicroRNA sequence codes for small extracellular vesicle release and cellular retention. 🤯 tinyurl.com/mr3zzfzv
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A pleasure to see that the @journal_nd special issue celebrating Terry Partridge’s contributions to muscle research and mentorship is out. content.iospress.com/journal… Thanks Raju for co-editing, @LochmullerLab, @JenniferEMorgan, Carsten and JND team for help and Pete Zammit for👇
Image from Jyoti K Jaiswal (@jyoti_k_jaiswal)
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A new study, co-authored by @ElsevierConnect's @mehmanib et al. reveals the #gendergap got wider, while paper submissions grew at the start of the #COVID19 pandemic: spkl.io/60184Ovtm #genderinresearch #researchworkforce #WomenInSTEM @squazzoni @LaStatale @PLOSONE
An illustration featuring the silhouettes of 6 'people', under a magnifying glass.

ALT An illustration featuring the silhouettes of 6 'people', under a magnifying glass.

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Together with Isabelle Richard @GenethonFr we describe a mouse model that mimics #LGMD2L muscle symptoms, offers insights into the basis of this disease, and test potential therapies content.iospress.com/article….
Image from Jyoti K Jaiswal (@jyoti_k_jaiswal)
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First R01 is being reviewed (or not reviewed) in study section today. It's totally normal to refresh my ERAcommons every hour, right?😬
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50 free copies of our article are available below!: Restoration of dystrophin expression and correction of #Duchenne muscular dystrophy by genome editing tandfonline.com/doi/full/10.… @tandfonline @UAlberta_FoMD #CRISPR

Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing

Introduction: Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that affects approximately one in 3500–5000 male births. Patients experience muscle degeneration, los...

tandfonline.com
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This. If I had continued in the cure mindset for my son’s Duchenne diagnosis, I would have wasted 7.5 years of his life so far being unhappy. Accept and adapt. Let it go. Move forward. Smile. #Duchenne #DMD #Disability
Henry Fraser (@henryfraser0) profile pictureHenry Fraser@henryfraser027 Jul 2021
We can only guess what would have happened on the roads we did not take. So there is no point dwelling on a past that cannot be changed. Don’t waste energy just hoping things were better. Acceptance allows you to move on. It allows you to live. Accept and adapt.
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Davi Mazala (@DaviMazala) profile picture
Davi Mazala@DaviMazala6 Jun 2021
Happy that our paper has been published! Human muscle stem cells are refractory to aging onlinelibrary.wiley.com/doi/…
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Delighted to co-host the new Research Topic "Genome and Transcriptome Editing to Understand and Treat Neuromuscular Diseases" with @RikaMaruyama_E @AlysonAFiorillo @MacGyver0282 and Dr. Duan in Frontiers in Genome Editing @FrontGenEditing frontiersin.org/research-top… #CRISPR

Frontiers | Genome and Transcriptome Editing to Understand and Treat Neuromuscular Diseases

Muscular dystrophies and other neuromuscular disorders are diseases characterized by muscle weakness and wasting that result from defects in the muscle itsel...

frontiersin.org
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