We have seen amazing drug developments. The life changing news of #orkambi & #kaftrio. But still we waited, as these weren’t for her. One day.
One day she will get something to give her more tomorrows for her genetic mix
So we got our letter from the #CF team saying our 6 year old isn’t eligible for the #Orkambi or #Symkevi. She will be when she’s 12. Hard to read. Six years seems like ages. @NICEComms isn’t looking at #Trikafta until 2021. These timings are too long for a progressive disease
The Bristol Childrens Hospital #CF team supports so many families in so many ways. We see them more often than we see our own families. We shall be getting letters about potential impact of the #Orkambi @VertexPharma deal for our children in the coming weeks.
We are so happy for the #CF community. This is the face of a girl who has been given a brighter future. Almost. #Symkevi licensed for 12 & over. We shall wait until she gets it. Thank you #facesofCF
Constant campaigning & seeing what is right & moral #Orkambi
Dum Spiro Spero
She also wants children. She has normal dreams. To know there are drugs our there like #Orkambi & watch new drugs being announced with no access to them in England is devastating & cruel. Please @MattHancock @VertexPharma @NHSEngland - please resolve this now
An important meeting is taking place today between @NICEcomms @MattHancock @VertexPharma - I really hope beyond anything this deadlock is resolved and patients have access to #Orkambi & #PrecisionMedicine. Families are going through psychological torture & people are dying 🙏🙏🙏
Drugs like #Orkambi are more than just medicines. They restore hope. This is what it’s like being told your child has the condition. Dum Spiro Spero.
‘We weren’t expecting you, but you are here now. Cystic Fibrosis – The Diagnosis’...
The Guardian view on new drugs: high hopes, higher prices
#cysticfibrosis #Orkambi www.theguardian.com/commentisfree/2019/feb/0...
Of my followers, statistically 1:25 that’s a 106 of you will be carrying the #cysticfibrosis gene - many blissfully unaware until you have a child born with it. That’s why I’m going on about #Orkambi & access to #PrecisionMedicine - it affects more of us than we realise.
What an amazing advance analysing the #genome. What is #PrecisionMedicine? learn.genetics.utah.edu/content/precision/intro/
The cost/benefits of such interventions eg #Orkambi cannot be understood using traditional models. This is the future, it is now. Please @MattHancock...
My 7 year old on holiday talking about his sister ‘I forgot she has cystic fibrosis yesterday’. How heartbreaking is this. He lives with the constant worry too. #PrecisionMedicine #Orkambi affects families too. Include their #QALYs & cost becomes a no-brainer @NHSEngland
For the U.K. to not provide access to #CysticFibrosis drug #Orkambi & other precision medicines is so demoralising, frustrating and life ending for many patients with CF. People are literally begging for this drug...
Up early. Written to MP @darrenpjones about access to #orkambi. Edited my book chapter. Drank tea and snuggled kids #noexams
Help me & @cftrust fight for access to #Orkambi & #Kalydeco . Sign the petition today! http://bit.ly/Orkambi
