It’s #EBAwarenessWeek - spread awareness about EB a rare skin disease that causes extremely fragile skin that blisters and tears with any friction.
Learn more and show support of #EBAwarenessWeek from Oct 25-31: https://t.co/OMBUMtWQeP
This is an incredibly important piece of news 4 investors of cell & gene therapeutics. CGTx, particularly in rare disease, have a much greater chance of approval than other drugs. Durable treatments & curative therapies are getting approved at much higher rates.
New analysis from @TuftsMedicalCtr NEWDIGS shared last week at #CGMesa23 shows that cell and gene therapies are substantially outperforming other, less targeted treatments in the clinic.
@FierceBiotech covers what the new data means for the sector.
https://t.co/ObPZUGsvk5
This is a very important milestone for the EB community.
debra of America Launches Registry for Individuals and Families Diagnosed with "The Worst Disease You've Never Hea... https://t.co/DThzKpqmnh
@debraOfAmerica is hosting another great EB Community Regional Meetup for individuals with EB and their families. Each event is an educational and social gathering to stay informed and connect with others in the EB community.
To learn more, please visit: https://t.co/0mIoDxCk46
@AaronBlank Go electric. While i love my Tesla there are now other viable options. Rivian SUV is nice & next year, Jeep is coming out w/ what looks like a nice one. In 6 months w/ the Tesla i’ve saved a net $650 by charging instead of paying for gas for my previous car, an Expedition.
Today, in partnership with The Assistance Fund, debra launched a patient assistance program to defray the costs of out-of-pocket medica expenses associated with eb. Expenses like co-pays, and insurance premiums are eligible for help...
https://t.co/sEcWAgLF3U
Can we get Rafi’s video 250,000 views and 2,000 comments? She’s getting up there already, 80,000 views more than 600 comments since this morning. https://t.co/CCTofpsi7c
Thank you @CBSNews for highlighting the exciting FDA approval of VYJUVEK @KrystalBiotech & its impact on quality of life for individuals with DEB like 15 y/o Rafi, daughter to debra’s Executive Director Brett Kopelan. Watch below. #genetherapy
https://t.co/U0cp1by4DS
Amazing new today. Krystal Bio's topically applied gene therapy was approved by the FDA! This is the first drug approved to treat EB and it has proven to be safe and effective. It's going to change Rafi's life.
https://t.co/uvqr8eLBPp
I was honored this week to host the #RDDS keynote panel with these amazing advocates @LeahEDSCN2A@BrettKope & Simon Frost. each shared their stories & lessons learned as they chart the path to urgently needed therapies for their children's #RareDiseases. #nextgenerationadvocates