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 ...
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.
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
EurekAlert!18d
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 ...
Woman, 28, reveals strange symptoms dismissed as a UTI that were actually a deadly cancer
Gianna Cericola from Philidelphia said the first symptoms she noticed included a burning sensation when she went to the ...
Business Wire8d
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 ...
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 ...
Investing9d
Ultragenyx reports positive data on gene therapy for rare disease
Ultragenyx specializes in developing therapies for rare and ultra-rare genetic diseases and has a portfolio of approved medicines and candidates in development. The information reported is based ...
health.economictimes.indiatimes6d
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 ...
pharmaphorum19d
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 ...