Today, we're excited to announce we have initiated the global clinical development program for TRCN-1023, an investigational antisense oligonucleotide designed to restore UNC13A protein function for the treatment of #ALS. CEO @ercgrn shares insight below. https://t.co/ESpPgs9bsE
1 in 400 people around the world will be diagnosed w/ #ALS in their lifetime. Meet Trace - on a mission to expand the promise of genomic medicine for those w/ neurodegenerative diseases, re-establishing healthy communication between nerves & muscle cells. https://t.co/UdCt0R5upR
@MauriceOnTW Thank you! Agree, the number dosed must certainly be larger now.
I’m especially curious how many individuals have been intrathecally dosed with AAVs. Perhaps this reference breaks it out. 🤞
I'm excited to announce our most recent publication analyzing the genotype-phenotype relationship in Metachromatic Leukodystrophy (MLD).
https://t.co/5MIZHG3V1l
The Swimmers https://t.co/D09VwsxNTx — a remarkably moving film that captures the desperation, heartbreak, loss, love, luck, and triumph of Syrian refugees
That's my little sis:
The Philadelphia Department of Public Health announces the appointment of Dr. Kinnari Chandriani to the position of Director, Division of Chronic Disease and Injury Prevention. @PHLPublicHealth https://t.co/GbQXi5stBU
Camzyos approval a milestone for HCM patients and exemplar for translating human genetics -> medicines. How can we apply this to more diseases? A few insights:
The (wrong) train’s crew members, however, were awesome — they identified another train we could intercept in Stamford and called the crew of that train to arrange our transfer. @amtrak customer service phone operators could use a lesson from the crew on train #148.
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Unbeknownst to us, our @amtrak train was posted to the wrong track out of NY Penn station. Only after the train started to move did we realize we were on the wrong train. The official @amtrak response was totally unhelpful and unsympathetic.
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@Bob_Wachter Do you have data on how the virus is impacting younger children who aren't vaccinated, both in infection rate but also severity of symptoms? Reading a lot of worrying comments in local parent groups.
Happy to see this work published! While the project didn't end in quite the way we wanted, we learned some interesting glycosphingolipid biology along the way.
Excited to share our study in which we evaluate CGT inhibition to reduce GalCers and sulfatides for the treatment of Krabbe disease and MLD. #BioMarin#WUSTL <https://t.co/6461gINhO0>
A thread...
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Excited to share our study in which we evaluate CGT inhibition to reduce GalCers and sulfatides for the treatment of Krabbe disease and MLD. #BioMarin#WUSTL <https://t.co/6461gINhO0>
A thread...
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