News

New blood test screens for thousands of rare inherited diseases at once
A new rapid blood test for newborns could potentially detect genetic mutations linked to thousands of rare diseases all at ...
Medscape19h
World Health Assembly Adopts Rare Disease Resolution
On Saturday, May 24, the 78th World Health Assembly (WHA) voted to adopt a resolution to establish a global framework to ...
Geneticist Jim Wilson left UPenn behind, but not his dream of curing rare genetic diseases
Jim Wilson‘s pioneering gene therapy explorations have helped launch eight companies focused on translating his 40 years of ...
New test can rapidly detect thousands of rare genetic diseases: ‘Revolutionary to families’
A groundbreaking blood test promises to transform the diagnosis of rare genetic diseases in babies and children, offering ...
World-first CRISPR therapy could 'transform' treatment for rare genetic diseases, but key challenges lie ahead
Scientists have used personalised gene-editing therapy to successfully treat a baby with a rare genetic condition. Could this ...
Open Access Government21h
New blood test rapidly diagnoses rare genetic diseases in children and infants
The test applies to potentially thousands of different diseases, and it can even detect new ones by providing the evidence ...
Medical Marketing & Media12h
Genentech taps Lorde’s ‘Royals’ director for film on rare disease caregivers
Genentech’s short film draws on the experiences of multiple families caring for children with hemophilia A, seeking to ...
Daily Journal12h
Being seen: Greenwood family helping raise awareness about rare disease
The McLaughlin family have become advocates to raise awareness about NF. The realization began to sink in — he wasn’t the ...
Healio14h
FDA grants rare pediatric disease designation for Stargardt disease gene therapy
The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated ...
Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA
Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. The biotechnology said Tuesday that the Food and Drug Administration granted the designation ...
FDA grants Ocugen rare pediatric disease designation as voucher program remains in limbo
Local drug developers have sold their rare pediatric disease designation program vouchers for more than $100 million. The FDA ...
Ophthalmology Times17h
Ocugen’s OCU410ST receives Rare Pediatric Disease Designation for the treatment of ABCA4-associated retinopathies
OCU410ST has received Rare Pediatric Disease Designation for ABCA4-associated retinopathies, potentially allowing Ocugen to ...