Search results

FYI- There's an expanded access program (EAP) compassionate use program available for the new triple combo, VX-445 As far as I know, it's not limited to US patients. (I think Ireland and UK are included, but other countries may be eligible). It's difficult to get any information from...

Very exciting results announced today for VX-445 :) Sounds like they'll review more data at end of 24 weeks and decide between VX-659 and VX-445 From Vertex press release on 3/6...

For those who don't already subscribe to the CF Roundtable newsletter: The special winter CF Roundtable® issue on Organ Transplantation in CF will be out soon. Subscribe by January 26th to ensure that you receive it. It’s free! Please go to: www.cfroundtable.com or...

I'm guessing most have seen this already, but thought I'd share in case you missed it: Incredible Phase 3 results for VX-659!!!:) From Vertex Press Release on 11/27: Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung...

The CFF is hosting a virtual event for those with CF and their caregivers on Sunday, June 3, from 4-9:30 p.m. ET. There will be keynote speakers and small breakout sessions to connect with others and discuss topics such as Sibling Relationships, Living a Fulfilling Life with CF, New Diagnosis...

Enrolling soon :) I heard this study is expected to fill up quickly, so let your clinic know if you're interested...

From VRTX.com: February 21, 2018 Vertex announced the initiation of the first Phase 3 study of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation. The study will...

Opportunity to put your unused IV supplies, G-tube, extra neb cups, etc. to good use: We are collecting pediatric medical supplies and are connecting with local families and organizations in and around the Houston area so that supplies can be distributed to the children who need them...

From Boomer Esiason Foundation: In light of the recent events in the Gulf Coast region, we here at the Boomer Esiason Foundation are raising funds that will directly impact the lives of those families in the cystic fibrosis community who were affected by Hurricane Harvey. Our team will be...

In participants 18 years and older who have one F508del mutation and one minimal function mutation, some will receive VX-445, ivacaftor and tezacaftor, and others will receive placebo. In participants who have two F508del mutations some will receive VX-445, ivacaftor and tezacaftor and others...

In participants 18 years or older who have one F508del mutation and one minimal function mutation, some will receive VX-659, ivacaftor and tezacaftor, and some will receive placebo. In participants with two F508del mutations, some will receive VX-659, ivacaftor and tezacaftor and some will...

This study is for people 18 years or older who have two copies of the F508del CFTR mutation and have been taking lumacaftor/ivacaftor for at least three months. https://clinicaltrials.gov/ct2/show/NCT03140527?term=Proteostasis&cond=Cystic+Fibrosis&rank=2

https://www.clinicaltrials.gov/ct2/show/NCT03161808?term=Rare+Cell+Collection&cond=Cystic+Fibrosis&rank=1 This study will consist of a single visit where researchers will collect nasal cells and a blood sample. CFTR genotype will be confirmed and a repository of rare CFTR mutation specimens...

To be considered you must have- Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy. Discontinuation of Orkambi therapy must have occurred <8 weeks from the first dose of Orkambi. FEV1 ≥25% and ≤90% of predicted normal for age, sex, and...

Vertex Announces Acceptance of its Applications for Review of the Tezacaftor/Ivacaftor Combination Treatment in People with Cystic Fibrosis by the FDA and EMA -FDA grants Priority Review of the application and sets action date of February 28, 2018- -Applications supported by positive results...

LAUREN MELISSA KELLY SCHOLARSHIP PRESENTED BY: UNITED STATES ADULT CYSTIC FIBROSIS ASSOCIATION DEADLINE: June 30th 2017 WHO MAY APPLY: Adults with cystic fibrosis who are pursuing career certifications, associates, and bachelor’s degrees Award: $1,500- $2,500 Application and Instructions...

Last week, CFRI’s executive director, Sue Landgraf, met with Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the United States Food and Drug Administration (FDA) in Washington DC, to encourage that the FDA expedite the review and approval of new drugs that can...

From Sue Landgraf, Executive Director of CFRI: A new proposed replacement for the Affordable Care Act (ACA) has been introduced in Congress; the current version has more support than last, despite containing essentially the same provisions detrimental to the CF and rare disease communities...

https://clinicaltrials.gov/ct2/show/NCT03119649?term=glpg&rank=44 ENROLLING | RESTORE CFTR FUNCTION Phase 2a study of Galapagos GLPG2222 in adults with CF › DESCRIPTION: This study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of GLPG2222...

There will be a great Webcast on Saturday, April 22, 2017 for the CF community. Important information will be presented on new treatments for CF that are coming soon, health insurance options, assistance programs and strategies for living with CF. We hope you can join us for the whole Webcast...