We’re a few weeks behind in updating this information but some big steps have happened recently regarding the potential FDA approval for Zavesca. The US Advisory panel on Jan. 12th recommended approval! This snippet is from the article on Reuters regarding the breaking news: Doctors can prescribe […]
Read more
This is an email from Nadine Hill of the NNPDF. This is good news. If the FDA does say lets move forward that means more families might be able to participate because the drug would be FDA approved. All though this isn’t a cure it is one […]
Read more
Abstract: Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death.
Read more
By Jocelyn Kaiser ScienceNOW Daily News 5 November 2009 Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against […]
Read more
