Paris, France – With ECRD 2024 scheduled for 15 and 16 May in Brussels and online, EURORDIS’ session, Revolutionising Funding Strategies for Breakthrough Therapies in Rarer Diseases, will address this critical issue. This session will not only highlight best practices but also showcase examples of therapies developed through innovative funding...
people News
New York — Rumi Scientific (“Rumi”), a developer of therapies for rare and neurodegenerative diseases discovered using its high throughput organoid phenotypic screening platform, announced the appointment of Allen Fienberg, Ph.D., as Chief Executive Officer (CEO) and Chairman of the Board of Directors. “Our human organoid-based high throughput drug discovery...
SOMERVILLE, Mass. — Sail Biomedicines, Inc., a Flagship Pioneering company and leader in RNA-based programmable medicines, today provided an update on its research funded by the Cystic Fibrosis Foundation (CF Foundation), with preclinical data that suggest that Sail’s Endless RNA™ (eRNA™) may offer the potential to treat the 10%-15% of...
Jurassic Park actor Sam Neill was recently interviewed by The Guardianopens in advance of the release of his memoir, Did I Ever Tell You? Originally planned as a glimpse into his career, his life as a celebrity, and his farm in New Zealand, the book takes a dark turn in...
PENNSVILLE TWP. — Sandy McWilliams, a former Pennsville resident, will walk 700 miles this summer from her home in Kentucky to her mother’s house in Pennsville to raise awareness on progressive supranuclear palsy, a rare disease that her mother is fighting. PSP is a rare, fatal brain disease that McWilliams’...
Santa Susana High School graduate Sean Shrager doesn’t believe in giving up. Though he was diagnosed with Duchenne muscular dystrophy (DMD) when he was 3 years old, Sean has never let his disease become an excuse for not attaining his goals. In fact, it has only motivated him to work...
CHARLESTON, S.C. – A South Carolina mother who has been battling a rare genetic disease for years and lost her two sons to the disease is sharing her story as MUSC works to treat this rare disease. Living in Lake City, Chantel Bass is a South Carolina resident that has...
ARLINGTON, Texas – Scott Schliebner, MPH, a leading clinical strategist for drug development of rare diseases, is joining 2M Clinical Inc. on Dec. 1. Schliebner, who most recently was Senior Vice president for Scientific Affairs and Therapeutic Expertise at PRA Health Sciences, has a 25-year successful track record of providing...
When their second child was only a couple of months old, the Liews noticed that he didn’t seem to be growing and that his stomach was bulging. At first, they thought nothing of it but when little Liew Yong Tai was a year old, he was diagnosed with beta thalassaemia...
NEW YORK – Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, announced today the appointment of Margaret Dalesandro, Ph.D. to its Board of Directors. Dr. Margaret Dalesandro is an accomplished biopharmaceutical executive with over 30...