CheckRare

🚨2026 orphan drug PDUFA & FDA approval dates are here! Get the latest timeline and status for rare disease therapies: checkrare.com/2026-orphan-dr… #RareDisease #OrphanDrugs #FDA #PDUFA

Trofinetide is now available in a new oral powder formulation for the treatment of patients with Rett syndrome. Learn how Daybue (trofinetide) Stix change disease management for patients at checkrare.com/daybue-trofine… #CheckRare #RettSyndrome #RettSyndromeTreatment #RareNeurology #RareGenetic

🔎New Learning Center- Neuroblastoma Learn more at checkrare.com/neuroblastoma/ #CheckRare #RareDisease #Neuroblastoma #RareNeurology #RareCancer

New data highlights the potential of repinatrabit as a promising oral therapy for phenylketonuria, with adolescents achieving significant and sustained reductions in blood phenylalanine levels. As a first-in-class approach targeting Phe transport, these findings reinforce its potential to expand treatment options as it advances into phase 3 trials. Learn more at checkrare.com/open-label-ext… #CheckRare #PKU #RareMetabolic #ClinicalTrials

Systemic mastocytosis can be easy to miss, presenting as anything from a simple rash to fatigue or even unexplained anaphylaxis. Elevated tryptase levels can signal a systemic cause, not just allergies. Learn how earlier diagnosis and a multidisciplinary approach are critical to improving patient outcomes at checkrare.com/beyond-the-ras… #CheckRare #SystemicMastocytosis #RareAutoimmune #RareDisease

Today is PLGD-1 Awareness Day. Learn more about this often misdiagnosed disease and its management at checkrare.com/plasminogen-de… #CheckRare #PLGD1 #PlasminogenDeficiency #RareGenetic #RareOphthalmology

CheckRare is on site at #ASPHO2026 learning about Immunotherapy Unleashed: CAR-T Innovations and Vaccine Combos Taking on Refractory Solid/CNS Tumors. Stay tuned for additional conference coverage.

The Arms Wide Open Childhood Cancer Foundation’s mission is to fund less toxic therapies for children with cancer to improve quality of life and to give children battling cancer and their families hope during the most difficult days of their lives. Learn more about this inspiring organization and their initiatives at checkrare.com/arms-wide-open… #CheckRare #ArmsWideOpen #ChildhoodCancer #RareCancer

There is a new wave of innovation at the intersection of artificial intelligence (AI) and rare disease care. Learn more at checkrare.com/biomarkers-and… #CheckRare #AI #RarDisease #RareDiseaseManagement

CheckRare is on site interviewing at #AANAM. Stay tuned for conference coverage, including an interview with Sumaira Ahmed of Sumaira Foundation on their new study comparing rituximab to approved drugs for NMOSD. #CheckRare #NMOSD #RareNeurology

In this symposium from WORLD 2026, leading experts in Fabry disease discuss the evolving treatment landscape. Explore how rational drug design is translating into meaningful clinical impact, with a focus on pegunigalsidase alfa and its emerging role in patient care at checkrare.com/rational-desig… #CheckRare #RareDisease #FabryDisease #RareLysosomal #RareLSD

🔎New Learning Center- Narcolepsy: Recognizing the Signals, Reducing Diagnostic Delay, and Improving Patient Outcomes Learn more at checkrare.com/narcolepsy-rec… #CheckRare #RareDisease #Narcolepsy #RareNeurology

Tuan Vu, MD from USF Health, presents this morning’s Keynote: “What Neurologists Look for in Treatment Response” at the MGFA Annual Patient Conference #mgnpc2026

Samantha Masterson, President & CEO of Myasthenia Gravis Foundation of America, welcomes an audience of more than 500 patients and families at the annual National Patient Conference #mgnpc2026 at the Margaritaville Resort in Hollywood, FL.

Yesterday, CheckRare attended the Regional Cholangiocarcinoma Symposium at @MassGeneralNews and had the opportunity to tour the top lab in the world for bile duct cancer, led by Dr. Haley Ellis.

Prader-Willi syndrome presents with distinct but often underrecognized features. Early recognition is key to reducing diagnostic delays and improving management strategies. Learn more at checkrare.com/clinical-featu… #CheckRare #PraderWilliSyndrome #PraderWilli #RareEndocrine #RareGastrointestinal #RareGenetic #RareNeurology #RareDisease

Updates regarding the phase 2 PIONEER study testing avapritinib (Ayvakit) in patients with systemic mastocytosis were presented at the 2026 AAAAI meeting. Learn more about results and next steps at checkrare.com/results-of-the… #CheckRare #SystemicMastocytosis #RareAutoimmune #RareDisease

❗Tomorrow, April 7th at 1:00pm EST. Last chance to sign up for our Live CME Webinar on Lysosomal Disorders and the Heart with Ozlem Goker-Alpan, MD, and John Jefferies, MD. Click the link to register: us06web.zoom.us/webinar/regist… #CheckRare #LiveCME #RareDisease #LDRTC

PLGD-1 is caused by defects in the PLG gene and is characterized by the body’s inability to produce adequate quantities of plasminogen. Dr. Amy Shapiro provides an overview of this rare genetic condition at checkrare.com/type-1-plasmin… #CheckRare #PLGD1 #RareGenetic #RareDisease

=📢 March 2026 FDA Milestones in Rare Disease Care! A busy month for FDA approvals in a variety of rare diseases. Stay up to date with our 2026 PDUFA Dates and FDA Approvals page at checkrare.com/2026-orphan-dr… #RareDisease #FDAApproval #MedicalBreakthroughs #CheckRare

Options for Fabry treatment have expanded. This Fabry Disease Awareness Month learn about Elfabrio, a PEGylated enzyme replacement therapy. elfabrio.com/?gad_source=1&… #FabryDiseaseAwarenessMonth #RareDisease #FabryDisease #CheckRare