Discover and read the best of Twitter Threads about #ATTR
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$NTLA and $REGN report initial data from ph1 study of #ATTR for NTLA-2001. All patients across the two doses showed 92-94% TTR reduction at day 28.
#crispr #genomics #genetherapy #biology #biotech
#crispr #genomics #genetherapy #biology #biotech
At both dose levels, $NTLA-2001 was generally well tolerated. 2 patients reported transient infusion reactions, which was the only observed treatment-related adverse event.
$NTLA-2001 is an in-vivo administered #genetherapy.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.
It is one of the first few in-vivo #GeneTherapy being tested. Congrats to the $NTLA and $REGN teams.
Multimodality imaging in patients with HFpEF: an expert consensus document of the European Association of Cardiovascular Imaging #EACVI #MMI #HFpEF academic.oup.com/ehjcimaging/ad… @ESC_Journals @Cosyns @alessia_gimelli @ThorEdvardsen @VDelgadoGarcia @bogdan_popescu1 @JaeKOh2 
This expert consensus document from the #EACVI provides recommendations regarding how to determine elevated LV filling pressure in the setting of suspected #HFpEF and how to use multimodality imaging to determine specific aetiologies in patients with HFpEF @EACVIPresident
📌Mechanisms of LV diastolic dysfunction include impaired relaxation, attenuated restoring forces, and increased passive elastic stiffness that leads to elevated LV filling pressure
📌The presence of LV hypertrophy and dilated LA provides support for the #HFpEF diagnosis
📌The presence of LV hypertrophy and dilated LA provides support for the #HFpEF diagnosis
Preclinical and phase I data for $AKCA-TTR-LRx (second generation inotersen, ASO conjugated with GalNAc), currently being tested in CARDIO-TTRansform, the largest #ATTR #Amyloidosis trial to date. Tafamidis allowed without restrictions.
onlinelibrary.wiley.com/doi/epdf/10.10…
#cardiotwitter
onlinelibrary.wiley.com/doi/epdf/10.10…
#cardiotwitter
Cojugation with GalNAc increased potency by 20-30x, allowing lower dose, lower frequency, less AE
