Rare Disease Review

The Medical Journal That Anyone Can Understand. Leader in #rarediseases & #knowledgetranslation. #zebrastrong

[Rare Disease Review @RareDRev]

"I am immensely proud of the work we have done and the impact we have had on the rare disease community." https://t.co/X1Q5YRvj4X

[Rare Disease Review @RareDRev]

“The dream in time for anyone suffering from a rare disease is that their care would be based on evidence, on what is known about the condition and not just on symptoms that arise.”
https://t.co/hc0mTw4xXE
#raredisease #cure #hope

[Rare Disease Review @RareDRev]

“Our work indicates that fine-tuning the level of CDK6 influences this mechanism and could potentially improve the quality of life of MPN patients. This opens up the possibility of a completely novel therapeutic approach.”
https://t.co/6BhUK6ku09
#RareDisease #treatment #hope

[Rare Disease Review @RareDRev]

“Life is never made unbearable by circumstances, but only by lack of meaning and purpose.”  - Viktor Frankl
#RareDisease #inspiration

[Rare Disease Review @RareDRev]

“A life spent making mistakes is not only more honorable, but more useful than a life spent doing nothing.”  - George Bernhard Shaw
#RareDisease #inspiration

[Rare Disease Review @RareDRev]

For him the revelation opened doors rather than closed them. He understood his body. He was happy to talk about his experience with others. Eventually, he stopped coming to see me, too busy enjoying his new life of vitality.
https://t.co/ofrhXkNMcP
#raredisease #medicine #hope

[Rare Disease Review @RareDRev]

If full length antibodies injected succeeds in mitigating TDP-43 pathology, the next step will be to humanize the antibody and to test it in model systems. If all does well, it will take about 3 years before initial testing in ALS patients.”
https://t.co/ri4uNQ1iXJ
#RareDisease

[Rare Disease Review @RareDRev]

In Canada, it takes seven years on average to obtain a correct diagnosis and treatment plan, and this process takes a major toll on a patient’s well being.
https://t.co/LKNugIGL8K
#RareDisease

[Rare Disease Review @RareDRev]

"If you don’t like something, change it. If you can’t change it, change the way you think about it.” -  Mary Engelbreit
#RareDisease #inspiration

[Rare Disease Review @RareDRev]

The use of CRISPR to cure ALS is promising since no cure currently exists. Research should now focus on the shortcomings of CRISPR to prevent off-site mutations and immunogenicity.
https://t.co/6GYgaFr6Yo
#raredisease #ALS #CRISPR #treatment

[RareDiseaseFdn @rarediseasefdn]

A heartfelt thanks to @tomkmiec @fsorbara @JennyKwanBC & @ElizabethMay for welcoming all to #RareDiseaseDay on Parliament Hill #Canada4Rare Thank you for sponsoring @TakedaPharma it was a great success!

[Rare Disease Review @RareDRev]

“He who fears he will suffer, already suffers because he fears.” - Michel De Montaigne
#RareDisease #inspiration

[National Organization for Rare Disorders (NORD) @RareDiseases]

What an incredible way to close out #RareDiseaseDay 2019 in the United States! Thank you @EmpireStateBldg for lighting up New York City in honor of the biggest day of the year for 350 million people around the globe impacted by #rarediseases. #ShowYourStripes #ShowYourRare

[Rare Disease Review @RareDRev]

Happy #RareDiseaseDay from Parliament Hill! @rarediseasefdn @raredisorders @TakedaPharma @tomkmiec @fsorbara @JennyKwanBC @ElizabethMay #ShowYourRare #Canada4Rare

[RareDiseaseFdn @rarediseasefdn]

Please join us and #ShowYourRare for #RareDiseaseDay on Parliament Hill this Thursday!! (here is a throwback from the last time we were #ShowingMyRare in #Ottawa)

[CORD @raredisorders]

Join us for International Rare Disease Day on Parliament Hill
Thursday, February 28 @rarediseasefdn @RareDRev @rarediseaseday #Ottawa #Rarediseaseday

[Rare Disease Review @RareDRev]

"Fibrodysplasia ossificans progressiva (FOP) is an ultra rare genetic condition in which a second skeletal grows in the body as the child ages." https://t.co/vmdg3I8gFf
#raredisease #Awareness #hope

[Rare Disease Review @RareDRev]

"Mepolizumab is a targeted therapy that aims to reduce eosinophil concentration, the immune cell responsible for mediating inflammation in Eosinophilic Granulomatosis with Polyangiitis" https://t.co/5Si89F0bxZ
#RareDisease #Immunology #treatment #hope

[Rare Disease Review @RareDRev]

With the new day comes new strengths and new thoughts. - Eleanor Roosevelt
#raredisease #hope

[Rare Disease Review @RareDRev]

"Adeno-associated virus–mediated (AAV-mediated) CRISPR gene editing technology can eventually be used in the treatment of Duchenne muscular dystrophy, according to recent findings."
https://t.co/u2PNGh1OAL
#raredisease #treatment #hope