Patients and scientists: THIS IS NOT A DRILL. The White House is considering requiring open access to all taxpayer-funded research. Please sign to let The White House know that you strongly support this transformational initiative: oaintheusa.com #OAintheUSA

Epic Software trying to block rule that can give #RareDisease patients and others better access their health information & improve care. Thanks Isaac Kohane & Ken Mandl for the article in STAT bit.ly/2sX8Ney #EMR #EHR #HealthIT #DigitalHealth

Biotech leaders discussing #raredisease #genetherapy - "Expanding AAV Manufacturing" @NCATs_NIH. Best practices, challenges, opportunities, logistics, analytics Check out agenda & speakers and video available later events-support.com/events/NCATS_G… #precisionmedicine #aav #celltherapy

FDA releases six final guidances today on #genetherapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations. fda.gov/news-events/pr… #raredisease #viralvectors #CDMO

Neurogene expands NO-COST genetic testing for children with unprovoked seizure age 5 years to under 8 years & joins Behind The Seizure invitae.com/en/BehindTheSe… to improve diagnosis for patients with #raredisease Batten disease. #CLN5 #CLN8 #Battendisease businesswire.com/news/home/2020…

Paper covers #raredisease therapies - pros/cons for #genetherapy #celltherapy,small molecules,monoclonal antibodies, protein replacement therapy, oligonucleotides, drug repurposing. Also approval stats, patient engagement #precisionmedicine #AAV go.nature.com/3bxh7TF

Incredible that @UABnews has developed a drug used to treat select patients infected with the Coronavirus - both in the US and in China. This potential treatment is the result of research occurring in Alabama & could help save thousands around the world. uab.edu/news/health/it…

Rare Diseases Day is today on the rarest day in the calendar. Here are some of our #adcy5 warriors. #acy5 #RareDisease #precisionmedicine #cdmo

Device hackers and engineers: with the impending ventilator shortage, there is a collaborative effort to create an open source ventilator and other devices: projectopenair.org Dana M. Lewis | #OpenAPS 🤖

Neurogene is focused on developing life-changing #geneticmedicines for patients and families affected by rare neurological diseases. We are excited to join Twitter to highlight company updates and share information about the devastating diseases we are focused on addressing.

Neurogene is proud to launch the LivingBatten Facebook page, a community dedicated to connecting families with resources and information about #BattenDisease, with a focus on subtypes CLN5 and CLN7. Visit facebook.com/livingbatten to follow us on Facebook!

Because subtypes CLN5 and CLN7 are rare, much of what we know comes from families and individuals living with #Battendisease and molecular research on the genes. Visit bit.ly/3hj01vj for more information.

😢

reuters.com/article/us-usa…

Bertrand would have been 13 today. In his honor, I'm releasing a rough draft of The Algorithm for Precision Medicine on his site. It is a short summary of the all the science he taught me in the hunt for diagnosis and treatments. May it now help others. bertrand.might.net/articles/algor…

February 28 is Rare Disease Day 2021 and we celebrate the strength, determination, laughter, and love in the @ADCY5.org community. #raredisease #adcy5 #precisionmedicine #movementdisorder

Thermo Fisher viral vector mfg site, Cambridge, MA received a commercial license for viral vector manufacturing. 1st FDA approved CAR T therapy by Bristol Myers Squibb & bluebird bio for multiple myeloma #genetherapy #viralvectors #lentivirus #CDMO bit.ly/3vrLeVX

Good read from Neurogene on route of administration optimization for CNS disease #aav #viralvectors #genetherapy #raredisease #precisionmedicine #neurology neurogene.com/press-releases…

📢 Attention all genetic disease foundations: The most valuable asset you own is trusted access to families and the ability to assemble clinical trial cohorts, starting with single-patient IND studies that will de-risk selection of the primary endpoint for a NDA-enabling study.

Caffeine has changed our daughter's life, thus ours. This is the power of scientific collaboration - Paris Brain Institute & ADCY5.org #ADCY5 #movementdisorder #raredisease Paris Brain Institute Institut du Cerveau - Paris Brain Institute #genomics doi.org/10.1002/mds.29…