Gene variant could protect against ultra-rare seizure disorder, fruit fly research finds
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
EurekAlert!19d
JAX researchers alleviate symptoms of ultra-rare genetic disease
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy and bone marrow transplantation – to alleviate symptoms of multiple ...
technologynetworks5d
Tackling Rare Diseases With RNA Therapeutics
We are a discovery engine and have a pretty good track record for multiple indications including numerous neurological compounds in preclinical studies and even a couple in the clinic for ultra-rare ...
pharmaphorum20d
Ultra-rare disease drugs: has access in England just got harder?
Meanwhile, NICE’s system for ultra-orphan drugs ... webinar brought together some of the key stakeholders in rare disease market access to discuss the changes, and see how all sides can work ...
pharmaphorum2d
Rare disease drug Vimizim gets NICE 'yes' – but needs real-world data
is undoubtedly the biggest issue for Vimizim and other rare and ultra-rare disease treatments. BioMarin has already offered a confidential 'patient access scheme' (PAS) discount to the £394,680 ...
Hosted on MSN1d
Ultragenyx Q4 Loss Wider Than Expected, Revenues Increase Y/Y
Ultragenyx Pharmaceutical RARE reported fourth-quarter 2024 loss of $1.39 per share, wider than the Zacks Consensus Estimate ...
Acsh.org4d
Rare Diseases Are Neglected. The FDA Needs A Dedicated 'Center of Excellence for Rare Diseases'.
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Rare disease patients and advocacy groups appeal to PM Modi for sustainable funding for treatment
Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.
Business Wire9d
Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance ...