I will scream my lungs out till it fills this room

Today NHS England finally came to a deal with the pharmaceutical company Vertex that means the 90% of CF patients who have the most common CF mutations will have access (within 30 days) to the first drug that they will ever take that treats the underlying cause of the disease, rather than only its symptoms. Read more here. 

Orkambi, Symkevi and Kalydeco are the first iterations of these small molecule drugs that will limit disease progression by allowing the normal movement of salt and water between cells. These drugs are not a cure, and the long term positive impact of taking them is not fully known, only time will tell, but even newer iterations of these drugs (like Trikafta) are already known to have a more dramatic impact and should be licensed here soon too. Today’s deal should pave the way for these future treatments. For children born today, the hope is these drugs will halt progression before the irreversible damage to their lungs ever occurs. 

This news (after 4 years of intense and passionate campaigning by people with CF and their families) is long overdue, and very, very welcome. 

Isaac is not a candidate for this drug, or others in the same pipeline. Unfortunately he is in the 10% minority that this drug will not work for (see blog below which explains why). But there is other research ongoing and pending clinical trials that may work for his rare mutation, as well as other drugs that are not mutation specific; but these are frustratingly a few years behind. We need to keep him well until these are available, so we continue to fight the progression of this shitty disease. 

If the momentum behind the research seemingly slows, then you will hear my screams. #don’tforgetthe10% 

Today is a very happy day. Next week, we start IV antibiotics again in the next round against the bastardly bugs camping in his lungs x 

I will light the match this morning so I won't be alone
Watch as she lies silent
For soon light will be gone
Oh I will stand arms outstretched pretend I'm free to roam
Oh I will make my way through one more day in hell
How much difference does it make?
How much difference does it make yeah
I will hold the candle till it burns up my arm
Oh I'll keep takin' punches until their will grows tired
Oh I will stare the sun down until my eyes go blind
Hey, I won't change direction
And I won't change my mind
How much difference does it make?
How much difference does it make?
I'll swallow poison, until I grow immune
I will scream my lungs out till it fills this room
How much difference?
How much difference?
How much difference does it make?

How much difference does it make?

Pearl Jam. 93.