#MDA celebrates the U.S. 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: mda.org/press-releases…

Positive news for boys with #Becker #MuscularDystrophy : Vamorolone improves disease progression with fewer side effects than current treatments. Read more from The Heier Laboratory, Alyson Fiorillo, PhD & the Center for Genetic Medicine Research. ⬇️ …ovationdistrict.childrensnational.org/new-becker-mus…

Congrats to Jyoti K Jaiswal #JamesNovak & colleagues for their interesting new #CellDeathDiscovery paper: Altered muscle niche contributes to myogenic deficit in the D2-mdx model of severe DMD. tinyurl.com/y3za3cd2

Great team effort led by a very talented post-doc Nikki McCormack. We find 1) vamorolone shows efficacy in our bmx mouse and 2) demonstrate that vamorolone reduces dystrophin-targeting miRNAs and increases dystrophin in skeletal muscle and heart!

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 Muscular Dystrophy Association Parent Project Muscular Dystrophy (PPMD) NIH NHLBI iScience journal
cell.com/iscience/fullt…

Our co-founder Alex Johnson and her son Jack appeared on BBC Breakfast this morning to talk about Jack's journey down to London for the King's Coronation! 👑 Joining Jack

For male academics (who are 4x more likely to have a partner at home doing domestic care), the start of the COVID pandemic was a publishing boon. Of the 45 most prolific medical journal authors (60+ papers) during the 1st 18 months, just 5 were women.
bmj.com/content/381/bm…

Yass! Operation warp speed for rare diseases proposed by Peter Marks (CBER, FDA) #mda2023 NIH Funding Dr. Monica M. Bertagnolli take note rare disease community are sick of being patient .. your inefficient funding cycles and processes don’t cut it in the information age!

New Research: Editorial: Genome and transcriptome editing to understand and treat neuromuscular diseases frontiersin.org/articles/10.33… #GenomeEditing #CRISPR

It was a pleasure working with co-editors Rika Maruyama, Ph.D., Alyson Fiorillo, PhD, Christopher Heier, and Dongsheng Duan. Looking forward to more breakthroughs in the field! #ScienceAdvances #NeuroMuscularDiseases

Attending Gordon Research Conferences 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 🤷🤷

It's my great pleasure to present the first preprint from SheqLab: a tech-dev project that we think will transform RNA biology.

If you want to discover the cellular factors an RNA interacts with—practically any RNA, in nearly any context—I think you'll want to try O-MAP. (1/19)

Impressively, my 9-year-old answered correctly! #futurescientist

Hi #myotwitter , check out my latest selection of papers on the regulation of muscle mass, exercise and aging!

biomed.news/bims-moremu/20…

Kevin Murach
glynhowatson
Alyson Fiorillo, PhD
Bims: Biomed News

1st mouse model of Becker muscular dystrophy!! Excited our bmx paper came out this week, check it out here:
#Beckermusculardystrophy Alyson Fiorillo, PhD FiorilloLab Nikki McCormack Edgewise Therapeutics Muscular Dystrophy Association Cure BMD Muscular Dystrophy UK Parent Project Muscular Dystrophy (PPMD) #dystrophin onlinelibrary.wiley.com/doi/10.1002/jc…

Investigators at the Center for Genetic Medicine Research Alyson Fiorillo, PhD & Christopher Heier 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

I am so excited to share our newly published work on generating and characterizing the first mouse model of #Beckermusculardystrophy Christopher Heier The Heier Laboratory Nikki McCormack Edgewise Therapeutics FiorilloLab #duchennemusculardystrophy #dystrophin doi.org/10.1002/jcsm.1…

'universities are hiring people without paying salaries...investigators are [like] independent small businesses required to raise as much indirect costs for the institution as possible' goodscience.substack.com/p/interview-ge… via Gholson Lyon

My favourite study of 'how often does a family with small children get a respiratory infection?' is this one, from the USA:
doi.org/10.1093/cid/ci…
Children <5y have one of 16 common respiratory viruses detected in a nasal swab 50% of the year!