Another scientist whose work initially didn't get the recognition that it deserved - Victor Ambros was denied tenure at Harvard, even after publishing the seminal work that resulted in him receiving the Nobel Prize today!
The follow up work related to this manuscript was recently funded by @MDAorg and we are looking for talented post-docs and lab staff to join our team! You can email me at [email protected]. (3/3)
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)
Shout out to @TheHeierLab for another great collaboration. I also wanted to mention that my lab (@FiorilloLab) recently moved to @VCU to join the Center for Inherited Muscle Research led by @nickjohnson30. I am incredibly excited to be a part of this amazing group! (2/3)
#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
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
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. https://t.co/ZhoGX6u2fn
Great team effort led by a very talented post-doc @nikkimmccormack. 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 co-founder Alex Johnson and her son Jack appeared on @BBCBreakfast this morning to talk about Jack's journey down to London for the King's Coronation! 👑 @alljoinjack
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.
https://t.co/5CkjJfocqn
Yass! Operation warp speed for rare diseases proposed by Peter Marks (CBER, FDA) #mda2023@NIHFunding @NIHDirector take note rare disease community are sick of being patient .. your inefficient funding cycles and processes don’t cut it in the information age!