#EHJCVI 🫀 Prognosis in #Fabry disease - is there more to it than just the X count 🧬? Does LVH fully capture cardiac involvement in females with FD👩? 'Milder does not mean benign'⚠️The reaffirmed importance of sex-specific thresholds in FD ⏩ doi.org/10.1093/ehjc... #MedSky
#Dapagliflozin in CKD Patients with #Fabry Disease
#VisualAbstract by Elba Medina
www.kireports.org/article/S2468-0249(25)00...
#BOB #FABRY
headlines-world.com/advanced-sea...
#BARRY #DOCKS #RAILWAY #STATION
multi-search-tag-explorer.headlines-world.com/advanced-sea...
aepiot.ro
Long-term effectiveness and safety outcomes in adults with #Fabry #disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey onlinelibrary.wiley.com/doi/10.1111/... #hvhebron #malmin [Text complet]
#ASHG2025: Roberto Araujo, MD, PhD, Senior North America Medical Director for #Fabry and #Pompe disease at Sanofi, speaking Oct. 15 at the @geneticssociety.bsky.social 2025 conference in Boston.
#RareDisease #FabryDisease #PompeDisease #Sanofi #MedSky #RareSky
Maison à Stoumont, tableau d’Émile Fabry.
Choisis 20 tableaux qui t'ont marqué-e ou influencé-e – un tableau par jour pendant 20 jours, sans ordre particulier.
#BlueSkyArtChallenge #art #painting #Fabry
🚨 Postdoc opportunity!
Passionate about theoretical & computational biology? Help map the #fitnesslandscape of #Fabry disease!
Join @iimog.sigmoid.social.ap.brid.gy, @sabinefischer & @gokhalecs.bsky.social at CCTB, @uni-wuerzburg.de
🔗 [PDF] tinyurl.com/mvr76my7
#Postdoc
Tomorrow, #SangamoTherapeutics will release earnings. Key focus will be #Fabry deal/no deal
To wit, unheard of POSITIVE eGFR slope (n=33) at year 1. Doing better than normal ageing! Close to 100k US patients if non-classical included. No LFT elevations requiring steroids.
This got to be valuable
Well-Established Monogenic Causes of Cerebral Small Vessel Disease
via @aanmember.bsky.social #stroke #fabry #CADASIL #CARASIL #neurosky
🔬🧬 Fabry Connections at @icmabcsic.bsky.social brought together 50+ researchers, clinicians, and patient associations to discuss the future of #Fabry disease research and care.
A day of science, testimony, and collaboration — despite the preivous blackout!
📸 More: icmab.es/event-recap-...
$SGMO eGFR slope remains positive following 52 week analysis of full 32 patient data in #Fabry gene therapy trial; pathway cleared for BLA submission in early 2026.
finance.yahoo.com/news/sangamo... #Sangamo
Patiently aggressive in this #biotech tape. Bullish on regulatory and M&A from here.
Waiting for $SGMO #Fabry deal, proceeds to be poured into $VERV and $QURE.
Pushing the boundaries of therapeutics here at @edinburgh-uni.bsky.social If you want to know more about our work in the hub on #Fabry and #MPSIVA get in touch. #ML #engbio #rare #diseases
$SGMO based on this chart, Sep 12, 2024 cut-off, the last subject to reach critical 12-month mark in #Fabry trial (for filing for AA) is about now.
The STAC-BBB deal was only made (in haste) to tide them over to the #Fabry deal. In theory, it allows them to wait until midyear now to get the required 52-week data from all subjects and thus ask for a higher licensing fee.
$300M upfront minimum IMO. US co-co would be nice. #$SGMO
In a normal world, the #Fabry asset alone would give #SangamoTherapeutics a market value of $3B.
Strong science, but too little focus...at least in the past.
The #Fabry deal had been guided to mid-Q2. Midyear would be critical data read-out potentially enabling them to file. But data so far looking good. Only a few patients missing.
#SangamoTherapeutics
This April & May get free access to curated variant insights on 11 genes tied to rare diseases like #Fabry, #Parkinsons, #CysticFibrosis & more via Mastermind. Support research & care with high-quality data. Contact our team to learn more about our offerings today: www.genomenon.com/contact?utm_...
And much more! #PKD, tubulopathies, kidney development, kidney fibrosis, mucolipidosis, #transplantation, #Fabry, structure and function, #genetics ....
Thank you to the CheckRare for this video taped during the 2025 WORLD meeting in San Diego summarizing our FACYS gene therapy trial for Fabry disease. #fabry youtu.be/vWyJO6I7awY?...
Piling in more heavily into #SGMO, because:
1) STAC-BBB systemic AAV delivery to CNS: not only data look better than competition, validation through 2 separate deals;
2) #Fabry gene Tx does not require post-infusion steroids, unlike hemoA. Sustained expression, improved QoL.
#fabry #fabricius #fabrivonhilden #hilden #medizin #chirurgie #medicine #wundchirurgie #arzt #doktor #wound #buch #book #booktok #chirurgia #chirurgien #earlymedicine #antiquariat #antiquariatsolder #münster
$SGMO could there be synergy between Nav1.7 lead internal #neuropathicpain program and #Fabry which is subject to licensing?
Pain flagged as major issue people with Fabry disease struggle with by AI:
$SGMO expectations for #Fabry deal is $300M upfront. But if so, why would acquirer not try and buy the whole shebang for little more? CEO sounds tired.
Fabry Disease - an inherited disorder that results from abnormal deposits of a particular fatty substance in blood vessel walls throughout the body. #Fabry #FabryDisease
Learn more at fabry.org & fabrydisease.org
You may be rare, but you are not alone!
#rareDisease #rareDiseaseAwareness
Global Quantum Cascade Laser Market to Grow at 4.5% CAGR, Expected to Reach USD 617.93 Million by 2032
#SNSInsider #global #quantum #cascade #laser #fabry #perot #interferometer #greenhouse #gas #analysis #Insights #photonics #INDUSTRY #NEWS
A flurry of new reports in JIMD Reports in the last 24 hours, first off we have this:
Phenotypic variability and the gender paradox in the R363C variant of Fabry disease
Alison Leslie, et al
doi.org/10.1002/jmd2...
#fabry
