Just wanted to share this cool study I read this evening, scientific advancements in gene discovery is so cool!
Sequencing the genes of these five Boston Children’s patients — a cohort known as “long-term non-progressors”— the researchers found a set of rare and never-before-discovered genetic variants that might help explain their longevity and stable lung function.
Here is a link to the full article > Why do some patients live longer than others?
Sequencing the genes of these five Boston Children’s patients — a cohort known as “long-term non-progressors”— the researchers found a set of rare and never-before-discovered genetic variants that might help explain their longevity and stable lung function.
I know it's been a while since I've shared some new information that has been in the Drug Pipeline for CF. This first one isn't for Little Miss Khaos (her mutation is DDF508) but it is great news for those that use Kalydeco! You can learn more about all of the drugs going through studies here.
FDA Approves Ivacaftor for Five Splice Mutations
The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
Full Information Here
This second one is for her mutation!
Positive Early Study Results for Next-Generation CFTR Modulators
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
FDA Approves Ivacaftor for Five Splice Mutations
The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
Positive Early Study Results for Next-Generation CFTR Modulators
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
Cystic Fibrosis Awareness Facts
Here is a list of Cystic Fibrosis Facts that you can use in raising awareness. I will continue to add facts as I discover more, if you have any suggestions feel free to comment below and I will add it! You can see the facts below in a sharable format on our Facebook page.
What’s in a Mask?
Last week the CF Foundation released a statements to all clinics letting everyone know their official stance on a popular mask. The Vogmask. Many were left confused and annoyed because these adorable masks have made getting the angstiest of children to wear them with ease.
Two new Medical Advancements
I wanted to share two great reads regarding CF advancements that have happened recently.
Pick the Perfect Team Name
The hardest part for us when we decided to start our Great Strides team was picking a team name. I wanted to create a list to help others come up with team names, if you have any to add to this list feel free to comment and let me know.
Fundraiser Idea Database
This is your one stop place for fundraiser ideas to help raise money for your Great Strides team! If you have any ideas that have worked for you that aren’t on this list comment and let me know! It will continue to grow over time as I discover more.