Daraxonrasib Delivers Landmark Survival Gains in Previously Treated Metastatic Pancreatic Cancer
- May 7, 2026
The PBCers Conference
FDA Priority Review Accelerates Path for IMAAVY as First Treatment for Warm Autoimmune Hemolytic Anemia
Enveda’s Oral Candidate ENV-294 Shows Early Promise in Atopic Dermatitis
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Kidneys play a vital role in keeping the body balanced, but when inflammation from lupus affects them, the warning signs can sometimes go unnoticed until damage has already begun. Recognizing subtle changes, understanding potential risk factors, and staying proactive with regular monitoring can make a meaningful difference in protecting long-term health. Awareness, education, and early intervention matter.
#lupusnephritis #autoimmuneawareness #PatientWorthy
Some conditions are so rare, they go unheard of for far too long—but the people living with them are navigating something real every single day.
Cystinosis is a rare genetic condition where cystine builds up in the body and can damage organs like the kidneys and eyes. It’s lifelong, and early diagnosis and treatment can make a meaningful difference.
Today on Cystinosis Awareness Day, we’re recognizing the importance of visibility, understanding, and continued awareness for this community.
Because awareness isn’t just information—it’s how more people start to understand why this matters.
#Cystinosis #CystinosisAwareness #RareDiseaseOfTheWeek #RareDisease #RareDiseaseAwareness
Upcoming: Virtual Symposium (May 8–9)
The SAMD9/L Lab at St. Jude is hosting a free virtual patient-researcher symposium this Friday and Saturday. I helped organize it, and it’s an important step in building awareness and momentum in the research community.
If you know doctors or researchers in rare disease, please share. Or if you like to attend any portion of it please register here:
2nd SAMD9 & SAMD9L Scientific Conference
www.stjude.org
SAMD9 and SAMD9L syndromes are recently discovered rare multisystem disorders that can affect the blood, immune system, brain, heart, lungs, hormones, digestion, bones, skin, and more. This conference...
