@CheckRare

Leading publisher and learning platform focused on rare diseases. Rare Diseases Are Our Focus, Expertise, and Passion.

 New York
checkrare.com
Joined December 2016
  • Tweets14.5K
  • Following1.6K
  • Followers3K
1.7KMedia
New FDA guidance is opening doors for rare disease innovation. OS Therapies’ CEO Paul Romness discusses how the Plausible Mechanism Framework will affect the approval process for OST-HER2 for the treatment of osteosarcoma. The goal is to remove barriers and exercise regulatory flexibility to encourage scientific advances and the delivery of more cures and meaningful treatments for patients suffering from rare diseases. Watch the interview with @CheckRare: bit.ly/4sx2xTr $OSTX
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Meet Dr. Abhinand from Chennai — an SMA Type 2 warrior who turned challenges into milestones From living with SMA to earning a PhD in Bioinformatics & becoming an Assistant Professor, his journey proves strength isn’t in steps, but in courage. 💪 #SMAAwarenessMonth #LifeWithSMA
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Dr. Goker-Alpan and the LDRTC team are attending the 39th Annual MPS society Conference in Denver, August 7-9! Registration is now open! Linked below. na.eventscloud.com/ehome/mps…
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Our VP of Sales & Marketing recently hosted a fireside chat with Medical Publisher @CheckRare at #WorldOrphanUSA, a forum focused on advancing orphan drug development and enhancing access to life-saving therapies for #raredisease. #WHIMsyndrome #RareResolve @OrphanConf
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On November 24-25, 2024, join us for GRIDS 2024 with leading experts at Capital One Hall in Tysons Corner, VA, to explore the latest in lysosomal storage disorders and rare genetic diseases. Register now: gridssymposium.org/grids-202…
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Today is LGMD Awareness Day! 💚 Help us Advocate - Educate - Celebrate limb-girdle muscular dystrophy. 💚Please share our post to help raise awareness. To learn more, visit LGMD-Info.org #LgmdAwareness #lgmdday2024 #TogetherWeAreStronger
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Describing a New Subtype of Distal Hereditary Motor Neuropathy Caused by RTN2 Deficiency New in @Brain1878 from the Inherited #Neuropathy Consortium (INC): academic.oup.com/brain/artic…

RTN2 deficiency results in an autosomal recessive distal motor neuropathy with lower limb spasticity

Maroofian et al. delineate a new form of autosomal recessive distal hereditary motor neuropathy (dHMN) with pyramidal features associated with RTN2 deficie

academic.oup.com
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Join @rarediseaseday in one month’s time for insights from accessibility experts and advocates on how you can make your website, social media and in-person events more inclusive! #RareDiseaseDay Register now! ✅ go.eurordis.org/42XmIr
A collage of photos showing accessible events and online experiences in front of a Rare Disease Day themed, purple, zebra background. Reads "Enhancing Digital & Physical Accessibility, 18 September 2pm (UTC+2)".

ALT A collage of photos showing accessible events and online experiences in front of a Rare Disease Day themed, purple, zebra background. Reads "Enhancing Digital & Physical Accessibility, 18 September 2pm (UTC+2)".

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In this recently published study based on research funded in part by IRSF, researchers applied a novel strategy to enhance RNA editing’s precision that proved successful in restoring normal MeCP2 in the brain of Rett mice. Read the article here: nature.com/articles/s41587-0…
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Our industry-leading speaker lineup for #WorldOrphanUSA keeps getting better and better! Today we're excited to share the addition of Ramaiah Muthyala, Professor/Director, @UMNews; President & CEO, @rarediseases_in🎤 Register as a group & save up to 50% – tinyurl.com/ye2x5dak
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Today is #throwbackthursday to kick us off we have Rake Garden Centre who wore it blue in May and held a cake sale in honour of Shelly Reid's daughter who has an MPS condition raising £162.46 thank you to everyone who donated we really appreciate your support! 💙 😊
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Celebrate World FSHD Day on June 20 with an Orange Slice Selfie! Head to our website for post ideas, suggested text, and a full toolkit of resources! 🧡🍊 fshdsociety.org/world-fshd-d… #CureFSHD #WorldFSHDDay #Neuromuscular #FSHD #RareDisease #FacesOfFSHD
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New research from the #LysosomalDisease Network (LDN): ✅ Exploring Management Approaches for High-Sustained Anti-rhGAA IgG #Antibody Titers in Patients with #PompeDisease Listen and read more: rarediseasesnetwork.org/news…
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Today is #RareDiseaseDay and we are joining @rarediseaseday to raise awareness for 300 million people worldwide living with a rare disease and for their families and carers. Take part in a free webinar on 'Patient Centred Research', 12.30-1.30pm: buff.ly/489x2DU
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Today is #RareDiseaseDay - a day to come together to raise awareness of rare diseases. Duchenne muscular dystrophy (DMD) is a rare genetic disease that causes muscle weakness and wasting. It's the most common and severe form of muscular dystrophy.
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Today is Rare Disease Day - a day for coming together to raise awareness of all rare conditions. Dravet Syndrome UK advocates for one of the many rare and complex diseases affecting families worldwide. #RareDiseaseDay #DravetSyndrome #DravetAwareness #StrongerTogether 1/2
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We can't wait to see landmarks like the Eiffel Tower, Empire State Building, Benjamin Franklin Bridge and more #LightUpForRare tomorrow! Share your photos of landmarks illuminated for #RareDiseaseDay & see how else you can get involved in the celebration: bit.ly/2BrULms
Text reading "Light Up For Rare This Rare Disease Day February 29" and "Learn more at rarediseaseday.us" with NORD and Rare Disease Day Logos and 3 skylines in purple, pink, and blue.

ALT Text reading "Light Up For Rare This Rare Disease Day February 29" and "Learn more at rarediseaseday.us" with NORD and Rare Disease Day Logos and 3 skylines in purple, pink, and blue.

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Did you know? 70% of rare genetic diseases start in childhood. #LaforaDisease is one of them! It onsets around puberty, with seizures typically as the first symptom. This #RareDiseaseDay, we hope you learn something new and amplify #RareDisease voices. 🦓 #FightLafora
Landscape rectangular graphic mostly comprised of text that says '70% OF GENETIC RARE DISEASES START IN CHILDHOOD! ARTICLE 'ESTIMATING CUMULATIVE POINT PREVALENCE OF RARE DISEASES: ANALYSIS OF THE OPHANET DATABASE, EUROPEAN JOURNAL OF HUMAN GENETICS (2019) #RAREDISEASEDAY RAREDISEASEDAY.ORG 29 FEB 2024'

ALT Landscape rectangular graphic mostly comprised of text that says '70% OF GENETIC RARE DISEASES START IN CHILDHOOD! ARTICLE 'ESTIMATING CUMULATIVE POINT PREVALENCE OF RARE DISEASES: ANALYSIS OF THE OPHANET DATABASE, EUROPEAN JOURNAL OF HUMAN GENETICS (2019) #RAREDISEASEDAY RAREDISEASEDAY.ORG 29 FEB 2024'

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🚨 IT'S RARE DISEASE DAY GLOBALLY! 🚨 Join us marking #RareDiseaseDay 2024! Spread awareness, share stories, and support those living with rare diseases. Together, we make a lasting impact! #ShareYourColours
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This #rarediseaseday, our dedication to supporting rare disease communities is stronger than ever. We are committed to supporting research and ensuring those facing the challenges of rare conditions have access to the treatments they need. #research #rarediseases #clinicaltrials
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