[ALA Client News] Novadip recruits first pediatric patients in pivotal phase 3 trial of tissue regenerative treatment NVD003 in congenital pseudoarthrosis of the tibia

ala.associates/clinical/nov...

#biotech #orphandisease

🚀 #OrphaDev4Kids is launching a #survey for #researchers working with Cyanotic Congenital Heart Disease (CCHD). If U have experience with CCHD help us advance #therapeutic options for this #orphandisease.
📝 survey here: redcap.emedea.it/surveys/?s=E...
#EU4Health #medicaldevices

Rare Disease feed contains posts related to Rare disease awareness, advocacy, research and support.

Tags included: #raredisease, #rarediseaseawareness, #rarediseaseday, #showyourstripes, #orphandisease

A call to reclassify the delta hepatitis virus as an orphan ... : Hepatology Communications An abstract is unavailable.

Delta hepatitis (HDV) is deadly, neglected, and underfunded.

Our new article calls for HDV to be reclassified as an orphan disease—a key move to drive research, drug development & awareness. It’s time 🦠📣

🔗 journals.lww.com/hepcomm/page...
#HepatitisD #OrphanDisease #LiverHealth #GlobalHealth

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality ...

World’s first: Gene editing clinically deployed for breakthrough treatment of rare genetic disease led by @kiranmusunuru & Rebecca Ahrens-Nicklas www.nejm.org/doi/full/10.... #GeneEditing #CRIPR #OrphanDisease #CPS1deficiency #TranslationalScience

If you are a #researcher with experience on CCHD or working on #cardiacdisorders, help us advance #therapeutic options for this #orphandisease.

Access the survey 📝 here: 👇
redcap.emedea.it/surveys/?s=E...

It only takes 1️⃣5️⃣ minutes!

#OrphaDev4Kids #medicaldevices #rarediseases #EUHealth

Hello BluSky! Vida Life 501(c)(3) nonprofit bridging gaps for Rare Disease & Syndrome (RDS) families. We connect patients, medical professionals, and researchers globally. Think social feed, searchable directories, and translated messaging. Excited to connect! #RareDisease #Nonprofit #OrphanDisease

#RareIsNotRare when there is community!

Despite postponement of 2025 FDA-NIH #RareDiseaseDay remember there is power in representation.
&
Let’s use facts as fuel.

udnf.org/udnf-stateme...

www.fda.gov/news-events/...

#RareDiseaseDay2025 #UltraRare #UndiagnosedDisease #OrphanDisease

COVID and Tongue Sore: Symptoms and Causes | Ada Health COVID-19 and tongue sores: explore symptoms, causes, and treatments. Learn how the virus affects oral health and ways to manage discomfort with Ada Health.

ada.com/covid/covid-...
#ada.com
#COVID-tongue
pre-existing multiple complex #hyperimmunity , #Genetic, & #OrphanDisease conditions
•COVID-free several weeks now, this condition occurred at the resolution of other Sx.
• now recurring.
Info for #COVID & related signs, symptoms
•My mouth is on fire

🚀 #OrphaDev4Kids is launching a #survey for #clinicians, #researchers and #caregivers of people living with Cyanotic Congenital ❤️ Disease (CCHD).
📝 redcap.emedea.it/surveys/?s=E...
If U have experience with CCHD help us advance #therapeutic options for this #orphandisease.
#EU4Health #HealthUnion

Centers for Disease Control and Prevention

@jeremyfaust.bsky.social
Thank you immensely, Doctor, for this resource.
Current #COVID with multiple idiopathic complex autoimmunity issues, #OrphanDisease.
Recent cdc.gov survey re: vax & pre-existing conditions was phenomenal. I utilize nhi.gov routinely. #StopRFKJr at all costs. Dangerous. 🙏🏾

Big time
I believe if one were to take all people with #OrphanDisease Dx and put them together, we’re not that Rare as a group.
It’s discouraging sometimes to feel like our numbers don’t warrant funding for research & Tx options.

✨Discover Delta4's roots and the journey from bioinformatics breakthroughs to AI drug discovery. Tune into our latest podcast for insights and innovations in drug development:
pharmaventures.com/how-is-ai-ch...

#drugdiscovery #delta4 #ai #innovationinpharma #fsgs #orphandisease

HypoPARAthyroidism Education, Support, Research And Advocacy - Patty Keating - Executive Director - HypoPARAthyroidism Association, joins me on Progress, Potential, And Possibilities #HypoparathyroidismAssociation #PattyKeating #RareDisease #OrphanDisease www.youtube.com/watch?v=eI6t...

We identified 515 new potential disease subtypes previously unrecognized, potentially transforming treatment options for rare and orphan diseases. 🧬 #RareDiseaseResearch #orphannet
@RareDiseases
#RareDisease #orphandisease

29.02. – Tag der seltenen Erkrankungen (Rare Disease Day) – FOAMio

Den Beitrag findet Ihr in einer längeren Form auch im Blog unter foamio.org/29-02-tag-de... #FOAMio #FOAM #Rettungsdienst #Notfallmedizin #Medizin #Welttag #WorldDay #RareDiseaseDay #SelteneErkrankung #RareDisease #OrphanDisease #ShowYourRare #ShareYourColours

FOAMio on Instagram: "Heute ist der #RareDiseaseDay, also der Internationale Tag der seltenen Erkrankungen. Worum es sich bei Seltenen Krankheiten handelt, wie diese definiert und charakterisiert werd... 4 likes, 1 comments - foamioblog on February 29, 2024: "Heute ist der #RareDiseaseDay, also der Internationale Tag der seltenen Erkrankungen. Worum es si..."

Drüben bei Instagram findet Ihr ab jetzt den Beitrag zu heutigen #RareDiseaseDay: shorturl.at/iuY19 #FOAMio #FOAM #Rettungsdienst #Notfallmedizin #Medizin #Welttag #WorldDay #RareDiseaseDay #SelteneErkrankung #RareDisease #OrphanDisease #ShowYourRare #ShareYourColours