Can’t say enough wonderful things about Dr. Ignacio Pérez de Castro and his team 👏👏👏👏👏 #ThankYou

The faces of #raredisease children are on the Jumbotron in Times SQUARE from Beyond the Diagnosis #RareInTimesSquare #RareDiseasDay #CTNNB1 #rarediseasechat

Six years ago I was told to prepare to die. This is the improbable, inspiring, love-filled, hope-filled, true story about the extraordinary campaign that happened next. This is where you come in: please share this trailer with everyone so that we can bring hope to everyone.

TODAY is #RareDiseaseDay! 🧡💙 All around the world, the rare disease community is coming together to #ShowTheirStripes, raise awareness, and demand equity for people living with a rare disease. As a global community, we have a powerful voice! 📣

Happy #RareDiseaseDay #CTNNB1

Hi, my name is, Effie Parks. Welcome to the CTNNB1 community. ::When the #CP patients get a test and start rolling in with a precise diagnosis:: #GeneticTesting eurekalert.org/news-releases/…

Roses are red 🌹 Violets are blue 💠 Text a #RareDisease friend today 📱 And say, "I see you." 👀

Join Monica Dudley-Weldon Saturday, May 6, 2023, for a networking lunch discussing the topic of "Parents of Newly Diagnosed Rare Pediatric Patients". Renaissance Downtown Hotel Washington, DC 12:10-1:10pm EST It’s not to late to register! livingrare.org National Organization for Rare Disorders (NORD) #syngap1

Carmela's Buddy Bear Campaign to help children in hospital. If you have a child who has Muscular Dystrophy, please contact Lucy (mum) via email to be added to the waiting list to receive a sponsored Buddy Bear teddy. [email protected] Muscular Dystrophy UK L-CMD Research Foundation

Ignacio Pérez de Castro (Ignacio Pérez de Castro), from the Institute of Rare Diseases Research of Instituto de Salud Carlos III (ISCIII) presents his research on: 🧬✂️ 'Heterogeneous responses to the application of different gene therapy strategies on an Lmna-R249W mouse of LMNA-related congenital muscular dystrophy'.

Check out AustinOfTheDay! #TikTok tiktok.com/@austinoftheda…

🌞 Good Morning Rare Friends "A warrior believes in an end they cannot see and fights for it. A warrior never gives up. A warrior fights for those weaker than themselves." #RareDiseaseTruth

Don't forget, the International Limb Girdle Muscular Dystrophy Conference is this October 27-29 in Washington, D.C.! buff.ly/41b9WtC

Congrats!!!!

The Children's Heart Foundation is proud to fund the American Academy of Pediatrics Pediatric Cardiology Research Fellowship Award.❤ The award represents a tremendous opportunity for junior faculty in pediatric cardiology to gain research experience. bit.ly/CHF-AAP

This is me. I am a 42 year old father and husband living with a currently fatal disease, ALS. I tweet about ALS, advocacy, changing the world and my life. My tweets have been called inspiring and raw. I dare you to follow me. You won’t regret it.

#GeneticTesting Let's get the kids diagnosed sooner and lessen the odyssey and give them a better opportunity to get care and contribute to research. And all the adults!! Where they at?

Helping millions affected with rare diseases starts with awareness. I’m proud of C.D. Leganés for making this a priority.

It's Muscular Dystrophy Awareness Month. Here are a few photos to show you what happens to me and others with my very rare type of MD called LMNA-CMD. It only affects around 1 in a million babies. Muscular Dystrophy UK L-CMD Research Foundation Cure CMD

Great to join DuchenneMD’s annual benefit for research into treatment for Duchenne Muscular Dystrophy with Senators Collins and Wicker, Congressman Joyce, and Chef José Andrés 🕊️🥘🍳. We're working to support treatment for this disease and I'm excited to see what advancements are next.