Aggregated News
The Western world's first drug to fix faulty genes promises to transform the lives of patients with an ultra-rare disease that clogs their blood with fat. The only snag is the price.
The gene therapy for lipoprotein lipase deficiency (LPLD), a hereditary disorder that raises the risk of potentially lethal inflammation of the pancreas, is likely to cost more than $1 million per patient when it goes on sale in Europe this summer.
Rare or so-called orphan diseases are winning unprecedented attention from drug developers. More than a quarter of the 39 new medicines approved in the United States last year were designated for such conditions.
These are therefore exciting times for campaigners such as Briton Jill Prawer, who leads an LPLD support group, and others championing the needs of people with equally obscure illnesses.
Naturally, she is delighted by the arrival of the LPLD drug Glybera. "It's brilliant," said Prawer, a 50-year-old mother of three who has suffered all her life from LPLD.
Until now, governments and insurance companies have largely accepted prices that can run into hundreds of...
The gene therapy for lipoprotein lipase deficiency (LPLD), a hereditary disorder that raises the risk of potentially lethal inflammation of the pancreas, is likely to cost more than $1 million per patient when it goes on sale in Europe this summer.
Rare or so-called orphan diseases are winning unprecedented attention from drug developers. More than a quarter of the 39 new medicines approved in the United States last year were designated for such conditions.
These are therefore exciting times for campaigners such as Briton Jill Prawer, who leads an LPLD support group, and others championing the needs of people with equally obscure illnesses.
Naturally, she is delighted by the arrival of the LPLD drug Glybera. "It's brilliant," said Prawer, a 50-year-old mother of three who has suffered all her life from LPLD.
Until now, governments and insurance companies have largely accepted prices that can run into hundreds of...