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.
Warrior Wednesday 2020 Impact Report
Warrior Wednesday amplifies the voice of the cystic fibrosis community worldwide! As the ever changing landscape of CF brings new hope and new challenges, they stand united to empower our CF Warriors and their families.
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.
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.
Interview, part one
Interview, part two
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.
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..
Kalydeco Health Concerns
Some health concerns if your child is taking Kalydeco. Read more. Click here: http://www.fda.gov/Drugs/DrugSafety/ucm316693.htm Please check back or email us with any questions. info@roadmaptocf.org
Hepatitis C investigation at The University of Michigan
UMHS one of six Michigan facilities linked to New Hampshire hepatitis C investigation
The Michigan Department of Community Health has been investigating the employment history of a Hepatitis C-positive health care worker who was recently arrested in New Hampshire for allegedly obtaining injectable narcotics at hospitals there, and infecting patients with the Hepatitis C virus through used syringes. To read more please click below:
