ADCY5.org Community

Caffeine has changed our daughter's life, thus ours. This is the power of scientific collaboration - @ParisBrainInst & ADCY5.org #ADCY5 #movementdisorder #raredisease @ParisBrainInst @InstitutCerveau #genomics doi.org/10.1002/mds.29…

📢 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.

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

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

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

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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.

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!

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.

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

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…

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

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…

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

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

@BrentShaferCEO @Cerner Interoperability is key for the #raredisease world. Hope you can truly help make it happen. Thanks

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