CheckRare

Leading publisher and learning platform focused on rare diseases.

Rare Diseases Are Our Focus, Expertise, and Passion.

[CheckRare @CheckRare]

🚨2026 orphan drug PDUFA & FDA approval dates are here! Get the latest timeline and status for rare disease therapies: https://t.co/dKzcblxrPt

#RareDisease #OrphanDrugs #FDA #PDUFA

[CheckRare @CheckRare]

💡Rare Disease Spotlight: Prader-Willi Syndrome

Learn more about this rare disease with our Learning Center https://t.co/j7jLaa8Xit

#CheckRare #RareDisease #PraderWilli #RareGenetic

[CheckRare @CheckRare]

The #CheckRare team has been hard at work at #ASCO26 attending clinical data sessions. This was an exciting conference with a ton of presentations on the latest in #RareCancer!

[CheckRare @CheckRare]

Just one week until World Orphan Drug Congress USA 2026! Connect with leaders, regulators, and patient advocates to advance rare disease therapies, clinical trials, and patient access strategies from June 9–11 in Boston.

Last chance to register at https://t.co/ujuX0ew5fx

#WODC #WorldOrphanDrugCongress #OrphanDrugs #RareDisease #PatientAdvocacy

[CheckRare @CheckRare]

Join us in celebrating CAH Awareness Month this June!

CAH is a group of rare genetic conditions caused by lack of 21-hydroxylase enzyme needed in the adrenal glands, causing the body to not be able to produce adequate cortisol.

Learn more about this rare disease and its diagnosis, management, and research advancements at https://t.co/6DdroJ8t5g

#CheckRare #CAHAwarenessMonth #CAH #CongenitalAdrenalHyperplasia #RareEndocrine

[CheckRare @CheckRare]

June 6th is the 2nd World Collagen 6 Myopathy Day! We’re shining a light on COL6,
an ultra-rare, genetic 🧬, neuromuscular disorder, impacting the lives of thousands
of individuals. Learn more about COL6 by visiting https://t.co/2cjjWviKYO

#CheckRare #RareDisease #COL6Day #COL6Myopathy #RareGenetic #RareNeurology #RareMusculoskeletal

[CheckRare @CheckRare]

Join the World Orphan Drug Congress USA 2026 from June 9-11 in Boston! Connect with leaders, regulators, and patient advocates to advance rare disease therapies, clinical trials, and patient access strategies.

Register now at https://t.co/ujuX0ew5fx

#WODC #WorldOrphanDrugCongress #OrphanDrugs #RareDisease #PatientAdvocacy

[CheckRare @CheckRare]

Narcolepsy is caused by the loss of hypocretin-/orexin-producing neurons, a discovery made in 1999 thanks to Dr. Mignot’s research that led to advances in diagnosis and treatment. Although current pharmacotherapy is still largely focused on symptomatic treatment, investigational orexin-2 agonists may be the future of disease modifying treatments in narcolepsy.

Learn more at https://t.co/DzJoXQ7QFb

#CheckRare #Narcolepsy #RareNeurology #RareGenetic #RareDisease

[CheckRare @CheckRare]

Highlights from #AAN2026 are now live on our website! Learn about the latest research in rare neurology with leading physicians, advocates, and healthcare professionals.

https://t.co/74QF8bulOP

#CheckRare #RareNeurologyDisorders #ClinicalTrials #PatientAdvocacy #RareDisease

[CheckRare @CheckRare]

Join the World Orphan Drug Congress USA 2026! Connect with leaders, regulators, and patient advocates to advance rare disease therapies, clinical trials, and patient access strategies from June 9–11 in Boston.

Register now at https://t.co/ujuX0ew5fx

#WODC #WorldOrphanDrugCongress #OrphanDrugs #RareDisease #PatientAdvocacy

[CheckRare @CheckRare]

PLDG-1 is underdiagnosed, but with increasing awareness, and the availability of effective treatment, diagnosis and management of these patients can be improved today.

Learn more at https://t.co/w4RzsHQSzl

#CheckRare #PLGD #RareGenetic #RareDisease

[CheckRare @CheckRare]

Systemic mastocytosis is a rare and often “hidden” disease that can present with a wide range of symptoms. Because it can mimic other conditions, diagnosis is often delayed.

This overview highlights the complexity of the disease, its impact across multiple organ systems, and the importance of greater clinical awareness to support earlier recognition and diagnosis.

Learn more: https://t.co/sVi0z4HasN

#CheckRare #SystemicMastocytosis #RareAutoimmune #RareDisease

[CheckRare @CheckRare]

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 https://t.co/EKJINEdAyl

#CheckRare #RettSyndrome #RettSyndromeTreatment #RareNeurology #RareGenetic

[CheckRare @CheckRare]

🔎New Learning Center- Neuroblastoma

Learn more at https://t.co/EuxolpDqV7

#CheckRare #RareDisease #Neuroblastoma #RareNeurology #RareCancer

[CheckRare @CheckRare]

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 https://t.co/EJczJoBW7K

#CheckRare #PKU #RareMetabolic #ClinicalTrials

[CheckRare @CheckRare]

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 https://t.co/7qdJ7aZI0z

#CheckRare #SystemicMastocytosis #RareAutoimmune #RareDisease

[CheckRare @CheckRare]

Today is PLGD-1 Awareness Day.

Learn more about this often misdiagnosed disease and its management at https://t.co/z5CAPFZwKB

#CheckRare #PLGD1 #PlasminogenDeficiency #RareGenetic #RareOphthalmology

[CheckRare @CheckRare]

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.

[CheckRare @CheckRare]

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 https://t.co/UrBwBklyr9

#CheckRare #ArmsWideOpen #ChildhoodCancer #RareCancer

[CheckRare @CheckRare]

There is a new wave of innovation at the intersection of artificial intelligence (AI) and rare disease care.

Learn more at https://t.co/oIjRT12aZz

#CheckRare #AI #RarDisease #RareDiseaseManagement

[CheckRare @CheckRare]

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