Historically, research into rare diseases has been siloed and lacking in standardization. This has resulted in meaning overlaps, wasted time, and wasted resources, all of which delay potential breakthroughs for... read more →
The need for real-world evidence to inform therapies can no longer be denied. Gathering standardized feedback from patients where they live can give researchers richer details into the effectiveness of... read more →
https://www.youtube.com/watch?v=jRfPtBnur1s Building on the success of our most recent webinar on Patient-Reported Outcome Measures (PROMs), our August webinar will expand on the topic of PROMs, delving deeper into the realities... read more →
https://www.youtube.com/watch?v=Wg-Bx8H1-j4& Daniel Lewi (CATS Foundation), Ellen Haf Davies (Aparito), and Femida Gwadry-Sridhar (Pulse Infoframe) discuss their experiences using rare disease patient data to drive research forward. Streamed on 29 July... read more →
July 20, 2020 — Robert Iannone, Executive Vice President, Research and Development and Chief Medical Officer at Jazz Pharmaceuticals Narcolepsy is still a widely misunderstood and under diagnosed disorder. Sadly,... read more →
Melbourne, Australia, July 20, 2020: It is estimated half the Australian population will experience a mental health disorder during their lifetime and one in five of the population had experienced... read more →
https://www.youtube.com/watch?v=EKgieiPGnG0 Patient registries—Does your population need one?
https://www.youtube.com/watch?v=I8SbtXpktH8 Learn about the CDKL5 Registry, International CDKL5 Disorder Database, & Connect CDKL5, and how they complement research goals. Q&A Panel will follow. Panel: Dan Lavery (Loulou Foundation), Helen Leonard... read more →
Front and center with the COVID-19 pandemic era is this question: How can we enable research to move forward while keeping patients who participate in studies and clinical trials safe?... read more →
With so much information whizzing by us these days because of the COVID-19 crisis, and with so many people posting pictures of themselves in #quarantine or #selfisolation, or talking about... read more →