Don't miss today's webinar from CureDuchenne and ReveraGen BioPharma 4 pm ET/1 pm PT. Register here: bit.ly/2qS4AHB

I hope the TACT model will be used in all #raredisease to foster drug development. These review panels bring invaluable drug dev expertise to emerging fields which may not have much experience yet, and they de-risk funding in areas where resources are often scarce.

A study from FDA's Peter Lurie on how woefully often the sponsor company's press release does *not* match the FDA CRL: "Press release statements matched 93 of the 687 CRLs (14%), including 16% (30/191) of efficacy and 15% (22/150) of safety statements." tinyurl.com/te84t9h

Congrats to April Pyle and Melissa Spencer of MyoGene Bio and AskBio Inc. (AskBio) for being named finalists for the "Buzz of BIO"! People of #myotwitter, please vote for them below to ensure #duchenne, Limb girdle and Pompe get prominent exposure at the BIO Convention. bio.org/events/bio-int…

🎉 We are proud to announce a $1M grant towards a clinical trial evaluating vamorolone in children and adolescents living with #Duchenne. Learn more about this new venture philanthropy investment and the Canadian clinical trial here: buff.ly/2MOvIyY ReveraGen BioPharma

Yes! And take note of the expected budget size for #Duchenne: $3.84M for Translational Partnership Awards and $4.48M for Idea Development Awards. So this makes CDMRP one of the largest funding programs for #DMD research. #myotwitter cdmrp.army.mil/funding/dmdrp

Exciting announcement from Santhera - Full enrollment of vamorolone’s VISION DMD trial. Subject to positive results of this first 24-week treatment period, this could pave the way for a regulatory submission to the US FDA in the fourth quarter of 2021 cureduchenne.org/partner-news/s…

ReveraGen Receives $3.3 Million NIH Commercialization Readiness Pilot Grant for NDA Preparations for Vamorolone in Duchenne Muscular Dystrophy pharmaceuticaldaily.com/reveragen-rece…

We are proudly continuing our commitment to the #Duchenne muscular dystrophy community. Today, we announced the first participant was dosed in our Phase 3 investigational gene therapy trial for affected boys aged 4-7. on.pfizer.com/39apCUr

.Santhera and ReveraGen BioPharma announce 2.5-year Treatment Data with #Vamorolone in #Duchenne #DMD globenewswire.com/news-release/2…

💵💰Funding opportunity in #Duchenne!!💰💵DMDRP has a $10M budget for Idea Development and Translational Research Awards. Submit your pre-applications by Aug 11th cdmrp.army.mil/funding/dmdrp

Congratulations to RESI Boston Innovator’s Pitch Challenge finalist, MyoGene Bio! Co-Founder & CEO, Courtney Young, will pitch to a panel of #investors September 21-22. Join RESI to cheer on our IPC finalists: bit.ly/3zWHS0E

In the first matchup in the #ARPAHDash Proactive Health Focus Area Reza Sameni with "Passive-Brain-Audition" is competing against Leo Grady with "Microbiome For Health". In the second matchup Emily Raycroft with "CRISPR Powered Biosensors" is competing against Courtney Young

EXCLUSIVE: Early findings in gene therapy death suggest CRISPR was not the cause statnews.com/2023/04/26/mus… via STAT Jason Mast

Huge congrats to Courtney Young of MyoGene Bio for being named one of MIT MIT Technology Review 35 Innovators Under 35!!!! technologyreview.com/innovator/cour…

Congrats to MyoGene Bio CEO, Dr. Courtney Young, for receiving a Life Science Catalyst Award from Biocom California!

Breaking news! Vamorolone, the first treatment for all DMD patients regardless of ambulatory status has been approved in the UK! The drug (sold under the brand name Agamree) has been approved for the treatment of DMD in patients aged 4 yrs+ by the MHRA. duchenneuk.org/first-treatmen…

NCATS’ DRDRI Director Dominique Pichard, M.D., M.S., is moderating the 8th rare story of #RDDNIH! Eric Hoffman, Ph.D., of ReveraGen and Sharon Hesterlee, Ph.D., of Muscular Dystrophy Association are discussing how a public-private partnership brought vamorolone to the market.