Discover more: https://bit.ly/4753QQc
Seven years of data support Takhzyro as a preventive therapy that reduces HAE attacks and improves disease control and quality of life.
@angioedemanews.bsky.social
@angioedemanews.bsky.socialDiscover more: https://bit.ly/4753QQc
Seven years of data support Takhzyro as a preventive therapy that reduces HAE attacks and improves disease control and quality of life.
@angioedemanews.bsky.socialExplore the details: https://bit.ly/4AgSOVo
Columnist Natalie Sirota, who is dealing with multiple diagnoses, says her current plan is to take it easy and try to avoid known stressors.
@mgnews.bsky.socialYour voice matters: sharing your experience empowers others. 🎤 #RareDisease
@angioedemanews.bsky.socialDiscover more: https://bit.ly/3T5ZOE4
A once-daily oral therapy for preventing swelling attacks in hereditary angioedema is now approved in Japan for children ages 2 and older.
@franmartinezgr.bsky.socialBi-allelic GAD2 variants cause a rare developmental encephalopathy with early-onset seizures #RareDisease #Genetics #morbidgene www.sciencedirect.com/science/arti...
@angioedemanews.bsky.socialTake a closer look: https://bit.ly/4xVK9pP
The FDA has granted fast track status to onvuzosiran, an HAE therapy that blocks swelling attacks and is currently in Phase 3 testing.
@mgnews.bsky.socialHave you ever thought you understood something about someone, only to realize you were completely wrong?
🔗 Read Shawna’s column - https://bit.ly/4yEsoeQ
#HumanSideOfRare #RareDisease #MyastheniaGravis #MyastheniaGravisNews #Bionews
@angioedemanews.bsky.socialExplore the details: https://bit.ly/4gTZ51R
A real-world study found that preventive treatment cut monthly swelling attacks in hereditary angioedema by 84% over more than two years.
@mgnews.bsky.socialTake a closer look: https://bit.ly/46rAlrL
A CAR T-cell therapy led to sustained improvements in muscle strength and daily functioning in generalized myasthenia gravis, per a study.
#MyastheniaGravis #RareDisease #MG #MyastheniaGravisNews #Bionews
@biopartner.bsky.socialJoin @camrare.bsky.social on Wednesday 7 October for a packed programme of talks, panels and interactive workshops at #RAREsummit26 - exploring policy, care, health data, partnerships and innovation.
📍 In person or online
🎟️ www.camraredisease.org/raresummit26...
@rarectadvocate.comIdiopathic Pulmonary Hemosiderosis (IPH) causes recurrent, unexplained bleeding in the lungs, mostly in kids under 10, leading to iron-deficiency anemia and possible lung scarring.
Learn more: https://dub.sh/IPH1
You may be rare, but you're not alone!
#rareDisease #rareDiseaseAwareness
@gamuts.netMultiple congenital anomalies - hypotonia - seizures syndrome: #Radiology findings https://gamuts.net/x/35234 #RareDisease #FOAMrad #Gamuts
@nephrogenomics.bsky.socialSharkVNTyper 1.2 is out ! another step forward for screening MUC1 kidney disease.
#Nephrology #Genomics #RareDisease #ADTKD #MUC1 #Bioinformatics #OpenSource
@angioedemanews.bsky.socialExplore the details: https://bit.ly/4yBf62y
An infant with recurrent limb and eye swelling was diagnosed with idiopathic angioedema after testing ruled out lymphedema.
@angioedemanews.bsky.socialGet the scoop: https://bit.ly/4iuLkrz
A woman who took HAE treatment Orladeyo for a brief time early in her pregnancy gave birth to a healthy child.
@theakarifoundation.bsky.socialEn The Akari Foundation apoyamos iniciativas como el proyecto de ley AB 1887 de California, que busca reducir las barreras de los seguros médicos y facilitar el acceso a tratamientos para quienes viven con enfermedades raras.💚
#TheAkariFoundation #California #EnfermedadesRaras #AB1887 #RareDisease
@curegm1.bsky.socialGM1 gangliosidosis is most often inherited when both parents carry one changed gene. Learn more at curegm1.org/about-gm1 #GM1 #GM1Gangliosidosis #RareDisease
@franmartinezgr.bsky.socialBiallelic ABCA13 Loss-of-Function Variants in a Child With Neurodevelopmental Delay: A Case Report #RareDisease #Genetics #morbidgene onlinelibrary.wiley.com/doi/10.1111/...
@thebabaklab.bsky.socialThe @fda.gov has approved the first therapy for glycogen storage disease type Ia (GSDIa).
Genglycos (pariglasgene brecaparvovec-opnr) received accelerated approval for patients aged 8 years and older.
Link in the comments 👇
