Five months on, kidney patient Abi, 13, who suffers from rare Dense Deposit Disease
, is still waiting.
Astonishingly, Abi's illness - known as dense deposit disease
(DDD) - is too rare to be covered by the NHS England funding policy, but not rare enough to be considered an exceptional case that would allow access to expensive drugs that could help save her life.
Jenna Smith is a 29-year-old Seattle resident with a rare condition called Dense Deposit Disease
(DDD) which led to her kidney failure.
Smith, 25, was diagnosed at age 6 with DDD - dense deposit disease
- an autoimmune disorder that prevents the kidneys from filtering waste from the blood.
Also known as Dense Deposit Disease
, it is so rare he is the only child in Ireland to have it.
Data from Animal Models Show Control of Kidney Damage in Dense Deposit Disease
Abi suffers from dense deposit disease
and is currently receiving dialysis for ten hours a day.
Because of its MOA involving the complement system, a mechanism potentially conserved across numerous disease conditions involving the immune system, its potential is also being explored in dozens of investigator-initiated trials for other conditions, including kidney transplantation, macular degeneration, CD59 deficiency, dense deposit disease
and C3 nephropathy, and cold agglutinin disease.
There are two types of MPGN: MPGN type I and MPGN type II which is also known as Dense Deposit Disease
English health watchdog NICE last week reported that 70 per cent of worldwide patients suffering from Abi's rare Dense Deposit Disease
improved after taking wonder-drug Eculizumab.
In addition, we enrolled the first patient in our pilot study of CDX-1135 in dense deposit disease
and initiated expansion cohorts in our Phase 1 study of CDX-1127 in metastatic melanoma and renal cell carcinoma.
The 12-year-old, who could be dead in five years from the rare kidney condition dense deposit disease
, has been thrown a lifeline by Nice - the National Institute for Health and Care Excellence.