Potential CF Therapy Kalydeco (VX-770) Available to People with Critical Medical Need

An expanded access program for a potential new CF drug, Kalydeco™ (VX-770), is now available at participating clinical sites throughout the country for people with the G551D mutation who have highly limited lung function and may benefit from treatment.

Vertex Pharmaceuticals, Inc., the maker of Kalydeco (kuh-LYE-deh-koh), created this program for patients while awaiting review of the drug by the U.S. Food and Drug Administration (FDA).

The company is seeking FDA approval for Kalydeco in people ages 6 and older with at least one copy of the G551D mutation.

People with CF who may be eligible for this program and want more information should talk to their CF doctor or call the Vertex Expanded Access Call Center at 1-800-745-4484.

Kalydeco is an oral drug in development that targets the underlying cause of cystic fibrosis. Kalydeco was developed by Vertex with CF Foundation support and research input.

http://www.cff.org/aboutCFFoundation/NewsEvents/12-20-VX-770-Available-to-Those-with-Critical-Need.cfm

VX-770 Shows Hope for CF Patients.

The Cystic Fibrosis Foundation and Vertex Pharmaceuticals announced today that VX-770, an oral medicine in development that targets the defective protein that causes cystic fibrosis, showed promising results in a Phase 3 clinical trial.

Read the article using the link below.

http://www.cff.org/aboutCFFoundation/NewsEvents/2011NewsArchive/2-23-Phase-3-Study-VX-770-Shows-Positive-Results.cfm

Pulmozyme Access Solutions

Pulmozyme Access Solutions Co-Pay Card Program by Genentech

www.pulmozymecopaycard.com

This is a program designed to help you stay connected to the medicine you need through co-pay support.

Eligible Pulmozyme patients should:

• Be over the age of 18 or have a legal guardian over the age of 18 with commercial insurance.
• Have a coinsurance of more than $30 per month.

If you are having trouble with the cost of co-pays from your Pulmozyme, then you should check into this program.

CFRI Presents Getting A Good Night's Sleep

Register early to view Getting A Good Night's Sleep on Tuesday, January 11, 2011 (6pm PST/7pm MST/8pm CST/9pm EST) online LIVE.

This free presentation is a part of CFRI and Stanford's CF Discovery Series. Nanci Yuan, M.D., will answer questions about CF-impacted sleeping problems and will discuss ways to healthier, more effective sleep.

To register, you must create an account on UStream and search for CFRI LIVE. Click here for registration page. To attend the event in person, click here. Special thanks to Genentech for sponsoring this event! http://www.cfri.org/home.shtml

NACFC 2010 - An Interview with Dr. Michael Boyle

Did You Know? Boomer Esiason Foundation has a Lung Transplant Grant Program

This is from the Boomer Esiason foundation web site. If you are a transplant candidate you may want to talk to your social worker at your clinic about this.

BEF LUNG TRANSPLANT GRANT PROGRAM
While the cost of transplantation is typically covered by most insurance companies, travel and relocation costs are typically absorbed by the patient’s families. The BEF Lung Transplant Grants Program is designed to help families cover the expenses that are not covered by their insurance.

Possible expenses to the recipient and family include, but are not limited to: Patient and family transportation costs for evaluation, surgery, and clinic visits after transplant; and housing, food, and living expenses associated with relocation to the transplant site.

http://esiason.org/index.php/site/cysticfibrosis/transplantgrants/

Article: One Step Closer to a Drug Treatment for CF

“The normal function of a cell is to pass chloride ions across the cell membrane at a very fast speed,” Hwang said. “We know some signaling molecules elicit this reaction, much like a hand signals an automatic water faucet to dispense water. But in the case of cystic fibrosis, that signal is no longer detected by the mutated channel protein. Through some mechanisms we still don’t quite understand, malfunction of this channel protein eventually leads to bacterial infection in the lung, which is believed to be responsible for the most severe symptoms of cystic fibrosis.”


The most recent study found that manipulating the sensor of the channel protein can significantly rectify the malfunction of the mutated channel, thus opening the door to a drug design that may eventually be a “real cure,” Hwang said.

Read the whole story using the link below

HealthNewsDArticleigest.com