Siempre son los boygroups floperos los que hacen que un himno como DUx4 se vea corriente y sin ritmo, sinceramente a estas retardaditas les hace falta un golpe de realidad con una presentación real

This is even worse than the skz dux4 cover goodness gracious 😭

pero no conocían dux4 y así

Proud to have contributed to a study of the Tapscott lab on the role of DUX4 in cancer

$ARWR continuing to move full steam ahead with 3 newly published US Patent Applications today covering DUX4, Skeletal Muscle Delivery, and the almighty MMP7 trigger targeting IPF. These are majorly impressive write-ups of 352, 105, and 348 pages. #RNAi

Haematology images: B-lymphoblastic leukemia/lymphoma with DUX4 rearrangement
buff.ly/44KIy8R

$ARWR +7%🎉
久しぶりに自信満々のCCでした😊

PoCの取れた肺への質問が多かったですが、個人的には筋肉ターゲットのARO-DUX4のディールが近そうなことも好印象でした😎
もうHBVに振り回されてないで済みそう。

あとはAATとAPOC3を来年中にゴールまで持って行ってくれれば。
長期視点で投資します💪

$ARWR (NP)

$2.5B EV

Nice to see RNAi moving forward in another muscle indication in addition to DUX4 in FSHD

$RNA reached $1.8B market cap largely on promising initial data for its DM1 program before unexpected AEs derailed their AOC tech

Early POC data for TRiM could spark

#FSHD displays overlapping manifestations with the neurodegenerative disease ALS.

Scientists identified Matrin 3, whose mutation causes ALS and dominant distal #myopathy , as a cellular factor controlling DUX4 expression and activity.

Cell Reports | 📘 bit.ly/3EGSDaA

Check out our recent study on single nucleus RNAseq in FSHD myonuclei suggesting a heterogeneous response to DUX4. Human Molecular Genetics, ddad186, doi.org/10.1093/hmg/dd…. FSHD Leiden

👀 Do we have a new therapeutic approach for facioscapulohumeral muscular dystrophy ( #FSHD )?

💊 Dr. Davide Gabellini and team support a pivotal role of #WDR5 in the activation of #DUX4 expression identifying a druggable target in their latest study.

📖 bit.ly/3KQa5fl

Any guesses when $DYN FSHD program enters the clinic? Any negative readthrough to $RNA FSHD program which has data 2Q24? DUX4 KD expectations? Muscle case studies coming? $FULC

Today we announced the filing for clearance to initiate a Phase 1/2 clinical trial of ARO-DUX4, our investigational #RNAi therapeutic being developed as a potential treatment for patients with facioscapulohumeral muscular dystrophy. Read more here: fal.cn/3zWAE $ARWR

Developmental regulators drive DUX4 expression in facioscapulohumeral muscular dystrophy biorxiv.org/cgi/content/sh… #biorxiv_molbio

Join us for Andrew Smith’s dissertation defense!
Title: The New Cancer Stem Cell: DUX4 drives a transient totipotent program in an assortment of cancers
Lab: Tapscott Lab, FH
When: Monday, July 31, 1:00 pm PDT
Where: Fred Hutch Pelton Auditorium & Zoom (email [email protected] for link)

Congrats to Pete Hendrickson, MD, PhD, who has received a Conquer Cancer, the ASCO Foundation Young Investigator Award! Pete Hendrickson is the first Duke Cancer #RadOnc trainee to complete residency through the novel RORS track. He's currently exploring CIC-DUX4 sarcoma 🔗 radonc.duke.edu/news/spotlight…

Happening TODAY - Andrew Smith’s defense!
Title: The New Cancer Stem Cell: DUX4 drives a transient totipotent program in an assortment of cancers
Lab: Tapscott Lab, FH
When: Monday, July 31, 1:00 pm PDT
Where: Fred Hutch Pelton Auditorium & Zoom (email [email protected] for link)

Robert Grange, HNFE professor, was funded by SOLVE FSHD to work on a research project to assess skeletal muscle torque as a functional outcome measure in the TIC-DUX4 mouse model of Facioscapulohumeral Muscular Dystrophy (FSHD). 🧑‍🔬

Read more here ⤵️
tinyurl.com/4t7rxcaf

Don't miss it! ArrowheadPharma ($ARWR) is kicking off its summer series of R&D webinars on Thurs. May 23 at 2pm ET. Part I will feature Lawrence Korngut, MD who will discuss ARO-DM1 in type 1 myotonic dystrophy and ARO-DUX4 in FSHD. Register: brnw.ch/21wJmo8

📆 Reminder! The first session of our summer webinar series, focused on neuromuscular disease, starts now. Tune into the session to learn more about our ARO-DUX4 and ARO-DM1 programs: lifescievents.com/event/arrowhea…