#chylomicronaemia
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genomeweb.bsky.social
The UK's NICE has recommended making two RNA-based drugs for familial chylomicronaemia syndrome available to certain patients.
UK NICE Backs Arrowhead, Ionis RNA Drugs for Familial Chylomicronaemia Syndrome
UK NICE Backs Arrowhead, Ionis RNA Drugs for Familial Chylomicronaemia Syndrome
ebx.sh
October 2, 2026 at 2:38 AM
ema.europa.eu
New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome 👉 www.ema.europa.eu/en/news/new-...
April 24, 2026 at 10:23 AM
pharmaphorum.bsky.social
Patients in #England and #Wales with #raredisease #familialchylomicronaemiasyndrome (FCS) will soon have a new treatment option, after #Sobi's #Tryngolza was recommended for #NHS use by #NICE.

pharmaphorum.com/news/nice-ba...
NICE backs Sobi's Tryngolza for rare disease FCS
Patients in England and Wales with familial chylomicronaemia syndrome will soon have a new treatment option, as NICE backs Sobi's Tryngolza.
pharmaphorum.com
October 1, 2026 at 1:47 PM
jimd-editors.bsky.social
Our latest Shortcast describes not 2 successful pregnancies in patients taking Volanesorsen for Familial Chylomicronaemia Syndrome, with Dr Charlotte Dawson and Antonio Ochoa



Soundcloud: http://bit.ly/3ZqaP3g

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#pregnancy #raredisease #triglycerides
November 29, 2024 at 9:18 AM
gimopenjournal.bsky.social
Complex genetic landscape of familial #chylomicronaemia syndrome revealed in the UK population, with regional and ethnic variation and nearly half of FCS cases linked to non-LPL genes. bit.ly/3JjesT0 #GIMO #Hypertriglyceridaemia #AutosomalRecessive
August 14, 2025 at 9:11 PM
pharmaphorum.bsky.social
The importance of #China as a growth market for #pharma multinationals is underlined by the news that #Sanofi has paid $130 million upfront for Chinese rights to an #ArrowheadPharma drug under review for a disease associated with elevated #triglyceridelevels.

pharmaphorum.com/news/sanofi-...
Sanofi grabs Chinese rights to Arrowhead rare disease drug
Sanofi has paid $130 million upfront for Chinese rights to an Arrowhead Pharma drug for rare disease familial chylomicronaemia syndrome.
pharmaphorum.com
August 4, 2025 at 9:10 AM
longtail-news.bsky.social
Feed: "PharmaTimes"
By: Emily Kimber on Wednesday, May 21, 2025
Ionis shares phase 3 results for olezarsen in moderate hypertriglyceridaemia
The drug was recently approved in the US for familial chylomicronaemia syndrome
pharmatimes.com
May 21, 2025 at 6:50 PM
kghlibrary.bsky.social
MHRA Approves Olezarsen for Rare Lipid Disorder
MHRA Approves Olezarsen for Rare Lipid Disorder
The MHRA has approved olezarsen for familial chylomicronaemia syndrome, a rare disorder linked to life-threatening pancreatitis.
www.medscape.com
April 14, 2026 at 1:00 PM
rarerevinsider.bsky.social
In our 'in the thick of it' series, Action FCS Chair Jill Prawer shares the top three unmet needs for the rare community living with familial chylomicronaemia syndrome (FCS).
https://rarerevolutionmagazine.com/rare-insider/putting-you-in-the-heart-of-the-rare-community-action-fcs/

November 11, 2025 at 1:02 PM