Why we need your help

As we wait for the outcome on Friday, we thought we’d share Maddi’s heartwarming story.

Maddi was 11 years old with a lung function of 24%, under palliative care. She was on Insulin, Bipap, overnight feeds, many medications and not able to receive a transplant. She missed out on a lot of school and activities as her hospital admissions were so frequent.

After almost 2 years of Trikafta she has a lung function of 58%, no insulin, most meds gone, no blood sugar scanner, and this week her feeding PEG was removed! After having PEG feeds for 5 years, she’s now gained enough weight that this is no longer needed! She has officially become a pubescent teenager. Something I never thought would happen.

Without her compassionate grant of Trikafta I was told to prepare for the worst, to give her a quality of life and do what I have always done… to be there. But now she’s planning to be a teacher or a vet when she grows up! She’s back at school, joined a netball team and hasn’t had any admissions since starting Trikafta!

Maddi’s mum Kylie hopes that Trikafta is the beginning, for the majority of people with CF, to a brighter future and better health. She hopes that other children get the benefits of this drug and that families don’t have to go through what their family have experienced.

Maddi & Kylie, it's amazing to read your story and we're so happy to hear that you're doing so well! We're crossing everything that it's a positive outcome on Friday and those waiting will have Trikafta soon #YesToTrikafta

Meesha is fortunate to have been on Trikafta for the last 4 months, after being accepted on the compassionate program.

For the last few years she has been very unwell and missed most of grade 2. Her lung function dropped as low as 32% and she was 95% tube fed, not eating anything most days. She was being closely monitored by the transplant team, but was hoping Orkambi and Trikafta could help her avoid this for as long as possible!

She’s now a completely different person on Trikafta, her appetite is back and she’s stopped the day time feeds, playing full games of netball and has stopped 3/4 daily inhaled nebulisers. Her diabetes has improved and is on much less insulin and lung function is 56%, her highest ever!

In her words ‘I’ve been feeling incredible!! I have SO much energy and I’m doing things I haven’t done in years!

Now I can dance, do lots of cartwheels, jump on the tramp for a long time and not have a violent coughing fit, throw up or get so out of breath I can’t breathe. I can take deep breaths. I never knew I could do that or what it felt like!!!

I feel like I’m a different person. I’m not tired anymore and can play all day long.

Best news today is my lung function (fev1) is 56%!! This is the highest it’s ever been!!!!

I hope all people with CF get to feel like this!! ’

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Meesha’s changes are amazing, we’re so happy to hear she got access and that Trikafta means she can be a healthy kid again.

We hope the PBAC and Vertex have found a way to get Trikafta to those waiting so we all get the same chance at health as soon as possible.

Aaliyah is fortunate to have compassionate use of Trikafta. All of last year she was very unwell. She would finish hospital admission and a few weeks later, sick again. Her lung function dropped below 40% in June and she started on oxygen at night.

Trikafta has been a miracle for us. Since starting on Trikafta in February this year she has put on 8 kilos, no longer needs supplements!

Her lung function is 99% no more oxygen! She has joined a Basketball team and is excited for her future!

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Aaliyah’s results are astonishing, we’re so happy to hear she got access and improved so much! We hope those waiting get a similar chance at good health in the near future!

~ The difference a miracle drug can make ~

Mother’s Day is extra special for Morgan today, as thanks to the Vertex medications, she’s celebrating her first year with young Zale! In her words, Orkambi is the reason she made it long enough to have a baby, and Trikafta is the reason she’s still here after having a bubba. She’s gained an astonishing 35% lung function! Trikafta has meant she’s now too well for the transplant list and has been given precious time to watch young Zale grow up. Something she’s dreamed of since she was young!

It’s a special day too for Kylie, who has two young kids, Caprice and Rylan. 8 years ago Kalydeco transformed her health and helped her get above 40%, have 2 miracle babies and stay healthy for 6 years. She’s struggled over the last 2 years, dropping down to 28% and is now starting the transplant process. She’s been rejected from the Vertex compassionate program over the last year, and in her words, is wondering if you have to be dead to get it?

Kylie desperately does not want to leave her two kids without a mother - and we hope that Vertex will provide compassionate access without delay. Their approach, omitting this group from their compassionate program, is needlessly cruel and shortening the lives of those waiting.

Kylie needs Trikafta now so she can have better health and precious time with her loved ones. Today is a day to celebrate all the mums who work so amazingly hard to keep both themselves and their loved ones well, and we hope that soon, there will be a life-changing, special box of Trikafta on the way