#RareDiseasesNews:How can patients' actively participate in clinical research?
One of the most relevant answers is Patient Reported Outcomes (PROs).
Know more about these validated and invaluable tools that are becoming indispensable in clinical trials👩🔬👨⚕️
#RareDiseasesNews:Microsoft has developed a version of Windows 10 software that responds to eye motion - It's just incredible when technology promotes #SocialInclusion and creates #EqualOpportunities 👌👏
DisCover more HERE
#RareDiseasesNews: New collaborative research project focused on finding new therapies for #NGLY1 deficiency counts with pharma, academia/government and a patient association. 👏👩🔬👍#CDGResearch
More information HERE
#RareDiseasesNews: The FDA has issues its first ever approval for a #GeneTherapy. Exciting & positive news keep reinforcing the potential of this type of therapy for many diseases.
Hopefully, this is the first of many other approvals for many other diseases and patients! #CDG 👏
Find more details HERE
#CDGPublications:Have you read the recent CDG & Allies-PPAIN scientific paper on Cardiac complications in CDG?
We dare you to read it, share it with your GP and spread the word! Heart diseases occur in 20% of all CDG and cardiac-related complications are strongly associated with mortality in CDG.
Access the abstract HERE get a full copy by writing to firstname.lastname@example.org
#RareDiseasesNews:A new type of scientific publication has been born: The Registered Report and it might well be changing the way clinical trial are conducted and perceived. What do you think? 👩🔬👨⚕️🤔
Read all about it HERE
#RareDiseasesNews: Pfizer innovates in Rare Disease care by promoting education & activity tracking for hemophilia patients:
- Hemocraft™, a modification of the popular video game Minecraft®;
- HemMobile® Striiv® Wearable, a custom wristband that allows monitorization of daily activity levels and monitoring heart rate to measure intensity.
What do you think of these innovations? Learn more HERE
#RareDiseasesNews: Proof of how #RareDisease Patients & Professionals are responsible for gigantic medical progress! A must-watch documentary! #CDGResearch=HOPE💚!
#RareDiseasesNews: Drug reporpusing: A shortcut to cures for rare diseases?
Learn more about this topic HERE
#RareDiseasesNews:95% of all rare diseases still lack a treatment. Humans have exhausted all the natural resources on Earth and preserving the environment has to be a top priority for all. We need to get criative!
Is there a way to tackle both simultaneously? 💚🤔
Full text available HERE