Scientist-Dad-Humanist
Boston, MA
Joined March 2013
- Tweets534
- Following94
- Followers114
- Likes200
James McArthur retweeted
Swallowing challenges (dysphagia) in #DM1 are caused by changes in the muscles involved in swallowing. Weakness and myotonia can disrupt how food moves from the mouth to the stomach.
Awareness can support earlier evaluation and safer swallowing. #DM1SymptomSpotlight #MyotonicDystrophy
James McArthur retweeted
Today we announced that we are advancing PGN-EDODM1 into the highest dose cohort in the Phase 2 FREEDOM2-DM1 study following DSMB review.
Read our press release here: pepgen.co/4z81HQT
James McArthur retweeted
Today we announced our first quarter 2026 financial results and recent corporate highlights. Read our press release: bit.ly/3QWoAoP
It is stories like this that shake me to the core when I see the profound scientific illiteracy that grips the USA and it’s current government!!! Measles Took My Daughter. This Is What I Want Everyone to Know. nytimes.com/2026/04/21/opini… via @NYTOpinion
James McArthur retweeted
Today, PepGen announced data from its low-dose cohort of the ongoing CONNECT1 Phase 2 clinical trial in Duchenne muscular dystrophy. We will be hosting a conference call and webcast today at 4:30 pm ET to review the data. Click here for more information: investors.pepgen.com/news-re…
James McArthur retweeted
SCDAA is disappointed by the @nbcsnl skit about the recent historic approvals of potentially curative gene therapies for #SCD. SNL chose to cast a spotlight on this news with a tone-deaf skit that is distasteful at best and harmful at worst. Read more: buff.ly/3Rv3Uk5
I am very pleased to have Dr Howard Mayer join our board of directors to join with @PepGenTx improving the future for patients with devastating neuromuscular diseases.
investors.pepgen.com/news-re…
Working with the community makes us stronger and smarter as we advance EDO51 for people living with DMD, amenable to an exon 51 skipping Rx. EDO51 demonstrated 20-fold higher levels of exon 51 skipping when compared to published data with naked oligo Rx in humans.
PepGen is grateful for the insights shared by members of the DMD community at the recent Duchenne Community Advisory board held this past weekend in Amsterdam. For more info on Duchenne CAB bit.ly/3QwnM7g
James McArthur retweeted
We are pleased to announce that the FDA has cleared the Investigational New Drug Application (IND) to initiate the FREEDOM-DM1 Phase 1 study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1) in the U.S. investors.pepgen.com/news-re…
Come meet Dr Michelle Mellion @PepGenTx Head of Clinical Dev @CNMDOttawa and learn more about our CONNECT1-EDO51 clinical study in DMD patients and Freedom-EDODM1 clinical study in DM1 and the EDO technology that drives extraordinary delivery of Rx oligos!
James McArthur retweeted
#DYK that #DuchenneMuscularDystrophy is typically diagnosed between 3 and 6 years old?
David, who lives with Duchenne, and his parents recount the initial signs that led to his diagnosis.
Watch his full story: bit.ly/3OTlgpF
#DuchenneActionMonth #WDAD2023
Proud to be working with this team of passionate people @PepGenTx to create a better world for people living with DMD and DM1
Appreciate the opportunity to talk on the @Biotech2050Pod podcast about my journey through biotech and @PepGenTx journey to develop the EDO technology in #DMD and #DM1 to improve the lives of patients. biotech2050.com/episodes/tra…
James McArthur retweeted
Meet Hanna Zhang, a Research & Preclinical Development Scientist here at PepGen. Hanna’s work on the team is crucial to our mission to transform the treatment of severe neuromuscular and neurological diseases #PepGenPioneers
James McArthur retweeted
Earlier this June, our Head of Clinical Development, Michelle Mellion, participated in a BIO panel discussing safety and long-term dosability in gene editing. We're proud of Michelle and her part in PepGen’s work to deliver potentially transformative therapeutics to those in need
James McArthur retweeted
We are excited to announce that Jaya Goyal, EVP of Research and Preclinical Development will be presenting in a Flash Talk today at the 5thAnnual RNATx Symposium at 3:30 pm ET. For more information, visit bit.ly/3Xl6Qmb #RNATx2023
James McArthur retweeted
This #PrideMonth, hear from Mallory, a transgender woman living with #DuchenneMuscularDystrophy. She talks about her experience living with a condition that primarily affects males and how it helped her to understand more about herself. #Pride2023
James McArthur retweeted
PepGen Pioneers participated in #WalkToDefeatDuchenne to raise funds and awareness for #DuchenneMuscularDystrophy research. Our inspiration, @defeatduchenne founder John Davidson, first pushed his son Jesse, who lives with #Duchenne, across Ontario in his wheelchair in 1995.
James McArthur retweeted
May is #MentalHealthAwarenessMonth
Hear from Leslie, who lives with myotonic dystrophy type 1 (#DM1), about how she tapped into the power of the #RareDisease community to provide mutual emotional support.
James McArthur retweeted
PepGen was honored to present preclinical data at AAN this year. A link to our press release which details the abstracts presented can be found here bit.ly/42xZ8pV
