Podcast on accessible information during COVID-19 and the challenges that come with isolation. Both Molly (web accessibility consultant) and Rebecca (psychotherapist) live with Usher syndrome, a condition causing combined blindness and deafness.
Molly Watt (25) is becoming a household name in the world of accessibility in technology. She is a young, independent entrepreneur, living with Usher syndrome. Accessibility, she says, is “anything, but black and white".
It has been roughly two years since David Rodman became the Executive Vice-President Research & Development at ProQR to apply his knowledge of study design and genetic diseases on our pipeline for genetic eye diseases.
The work at ProQR would not be possible without the help from patient organizations and foundations. One of the main supporters is the Foundation Fighting Blindness, who generously provided funding for the clinical development of QR-421a.
At a glance, it’s hard to tell Maartje - mother of four - has Usher syndrome. Though she manages her life very well, it took her a long time to get to that point. “I have learned to be optimistic, but the somberness is always there."
New therapies are thoroughly tested to ensure safety and efficacy, but for a company like ProQR, which aims to treat small groups of patients with rare diseases, innovating within the system is essential. Jessica is trying to rethink clinical trials.
ProQR has a promising pipeline in ophthalmology programs that is built on RNA technology. According to Dr. Peter Adamson, ProQR’s (former) Senior Vice President Ophthalmology Franchise, RNA may reach the ultimate goal: to make blind people see again.
Interview with Dr. Ted Dryja, Scientific Advisory Board member. The pioneer in inherited retinal diseases joined ProQR to help push the organization forward. “I'm excited about what RNA technology may do for ProQR’s ophthalmology pipeline."
When watching Cristian, Silvia and Edoardo (age 6) and Beatrice (age 2) they look like the ideal, happy family. When asked, the parents insist they are, in many ways. The fact that Beatrice has Leber congenital amaurosis, doesn't change that.
Bart Klein was at the forefront of ProQR's Axiomer® RNA editing technology development. This platform technology makes it possible to use the cells own machinery to reverse mutations that cause a genetic disease. Read the interview.
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.