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 ...
pharmaphorum18d
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 ...
EurekAlert!17d
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 ...
Hosted on MSN1h
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 ...
pharmaphorum3d
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 ...
FierceBiotech22d
FDA delays decision on Stealth's 2nd shot at approval for ultrarare disease med
but we really persevered because we were inspired by the potential that we believe we've delivered upon to improve the lives of patients living with this serious, rare disease.” ...
health.economictimes.indiatimes5d
RARE DISEASES
According to Dr. L Swasticharan, Additional DDG with DGHS, if successful, this model could be extended to other rare diseases in India. Study finds how genetic diagnostics of ultra-rare diseases ...
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.
Yahoo Finance8d
Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing genetic medicines for rare and ultra-rare diseases. CRD is committed to accelerating the development of ...