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Cystic Fibrosis In The News

Learn about the latest CF medical advances, treatments and pharmaceuticals.

Doctors in Egypt. Music video. Great work

Stepping back in time, that’s what it feels like to see what these Egyptian doctors have to work with in regard to medications. They have 4 vests for all 600 individuals with CF. In the U.S. usually each person with CF has a vest in their home. There are no CF modulators here (no Trikafta), it’s a dream for them right now. But these doctors are incredible, working to save the lives of these young children with this viscious disease. They don’t have the basics like vitamins, most of the patients suffer from malnutrition. When you watch the video you can see the transcript below if you need it. I love all these incredible people. They are hero’s. They are CF warriors. They are my people. xo

Dr. Sally Raafat Ishak “In Egypt now things are a little bit better than they were before, the creon is now on the label by the Ministry of Health, but the amount is still not sufficient to cover the needs of each patient daily. The hypertonic saline is now available, but we have the 7 percent instead of three percent, which is now available in the market. We still give the patient, the drug, but no added vitamins are still not available. And definitely the modulators are still far away from Egypt. We hope that they will be available in the near future to all of our patients.” Dr. Osama Taha Amir, Professor of pediatric pulmonology Zaga Zig University “ Our patient life expectancy is very short, young age. What, looking at the patient in Europe and America that live, almost normal life, they get to forty, they are very healthy. We hope that our children get this chance. We need them to get the modulator, we need them to get pulmozyme, to get every medication available to them. We need them to get the vest for every patient, not one vest for all our patients, no, we need vests for every patient. To get the best life. To live normal life.” Dr. Dina Tawfeek Sarhan, Assistant professor of pediatric pulmonology, Zaga Zig University, “The vest you donated will help our patients a lot. We are excited to start to use it on one patient and send you a video. I think we will be delight and our patients and you also. As the nutrition part, we don’t know what to do, we have the basics. Laura Bonnell asks: “From the United States to here, you’re at such a different stage, right? Dr. Mahitab Morsy Hussein, “Yes, yes, we are way behind. We are looking for to serving our patients in a better way. We are starting here our center of Excellence. The sweat machine is here. One year ago, we have used it. We have diagnosed a lot of patients, and we are aiming for more care, more better care. I know this is way behind, like in the U.S. But we are aiming better for our patients, and they deserve it.




CF Dad, Mohamed. A hero.

Mohamed hates cystic fibrosis. We can relate. The Bonnell Foundation is grateful for his honesty. Mohamed’s 13 year old son is well thanks to the hard work and research by his Dad. You’ll enjoy his breathtaking honesty in this video.




 

CF Mom Izraa. A wonderful moment meeting my first Egyptian CF Mom

Meeting Izraa, as you’ll see in this video, took me right back to diagnosis day. I began crying as soon as Izraa asked me if her 7 month old son would live. We hugged and couldn’t let go. I can’t speak Arabic and she can’t speak English, but there was no language barrier for us. We speak CF Mom. The video is very emotional.




Cairo, Egypt, Day 2

I’m Laura Bonnell and I am in Egypt to deliver my daughters pediatric vests to the Egyptian CF doctors here. Check out the video.




Travel to Egypt

Laura Bonnell is in Egypt with Stacey Fogerty Brown, U of M Dietician. The pair met Dr. Nasr in Cairo, Egypt to deliver vests donated by Bonnell’s daughters. Bonnell also received vitamins from a CF Mom and Creon (enzymes). The donations quickly were used.




FDA Approves SYMDEKO (tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene

February 12, 2018 — Vertex announced that the U.S. FDA approved SYMDEKO (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the CFTR gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. SYMDEKO is Vertex’s third medicine approved to treat the underlying cause of CF. Vertex is ready to launch SYMDEKO and will begin shipping it to pharmacies in the United States this week.

Read the press release

Vertex treats the underlying cause of cystic fibrosis in people ages 12 and older like Jessica Franklin of Maryland with certain mutations in the CFTR gene. Click the play buttons below to hear Laura Bonnell speak with Jessica about how this new medicine will affect her.

New Stage of Triple Combination CFTR modulator. Impacts people with Delta F508

January 31, 2018 — Vertex announced the selection of two next-generation correctors, VX-659 and VX-445, to advance into Phase 3 development as part of two different triple combination regimens for people with cystic fibrosis (CF). This decision was based on positive initial Phase 2 data. Regulatory discussions are ongoing to finalize the design of Phase 3 programs. Upon completion of these discussions, Vertex plans to initiate the first Phase 3 triple combination program in the first half of 2018 to evaluate VX-659 in triple combination, and a second Phase 3 program in mid-2018 to evaluate VX-445 in triple combination.

Read the press release

FDA Approves New Cystic Fibrosis Treatment

Via CNBC

The U.S. Food and Drug Administration said Thursday it had approved the use of Vertex Pharmaceuticals’ Orkambi, a drug aimed to treat patients with cystic fibrosis, on patients who are at least 12 years old.

The company’s shares were temporarily halted, up 3.73 percent at $130.90, during late-morning trading for pending news ahead of the announcement at 11:47 a.m. ET. Trading started again around 3 p.m. ET, and Vertex closed the day slightly higher than its halt price.

“…The approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community,” Dr. Jeffrey Leiden, Vertex’s chairman, president and CEO, said in a statement.

With the approval, Orkambi will be available to about 8,500 patients in the United States, according to Reuters.

Cystic fibrosis is caused by a defective gene that disrupts the function of the lungs and digestive system, producing a build-up of thick, sticky mucus leading to inflammation and recurrent bacterial infections..

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