Poster presentation at 20th World Symposium: A paradigm shift in collecting RWD for GM2 gangliosidosis. Collecting real-world data (RWD) for... read more →
Every parent loves to watch their child fly like a butterfly and discover the world. Although five-year-old Ollie’s variation of... read more →
by Dr. Femida Gwadry-Sridhar Have you ever considered the connection between drug development for rare diseases and olive trees in... read more →
Shattering Rare Disease Registry Recruitment Targets with PIP-UK Real-world data from registries and other real-world evidence sources remain a fertile... read more →
A Collaborative Partnership to Develop the CDKL5 Registry CDKL5 Deficiency Disorder (CDD) is a rare developmental encephalopathy with epilepsy that... read more →
Timothy Syndrome Alliance and Pulse Infoframe are pleased to announce that the CACNA1C Community Registry is open for worldwide recruitment.... read more →
You would think that, after 22 years in the U.S. Navy, a veteran deserves a break. But like a baseball... read more →
The Myrovlytis Trust, BHD Foundation, and Pulse Infoframe are pleased to announce that the BHD International Registry is open for... read more →
By Dr. Femida Gwadry-Sridhar It is astounding when you think about rare diseases and then you hear about rare epilepsies.... read more →
Visiting a UNESCO World Heritage Site is an incredible experience. Last December, I visited Teotihuacán in Mexico. The pyramids in all... read more →
