ADCY5.org Community

Supports #raredisease ADCY5 gene variant population - patients, doctors, researchers. #ADCY5 #AC5 #adenylylcylcase

[ADCY5.org Community @adcy5]

Caffeine has changed our daughter's life, thus ours.
This is the power of scientific collaboration - @ParisBrainInst & https://t.co/hLd4DGqC85

#ADCY5 #movementdisorder #raredisease
@ParisBrainInst
@InstitutCerveau
#genomics

https://t.co/0S46qF7tnF

[Ethan Perlstein 1-to-N @eperlste]

📢 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.

[Steve Grossman @stevenjgrossman]

Good read from Neurogene on route of administration optimization for CNS disease

#aav #viralvectors #genetherapy #raredisease #precisionmedicine #neurology https://t.co/e7MFhNyWiv

[Steve Grossman @stevenjgrossman]

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
https://t.co/cTIJVycsw1

[ADCY5.org Community @adcy5]

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

[ADCY5.org Community @adcy5]

https://t.co/QGCWyeH8D8

[ADCY5.org Community @adcy5]

😢

[Neurogene Inc. @NeurogeneInc]

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
https://t.co/p7Dse4Sbbm for more information.

[Neurogene Inc. @NeurogeneInc]

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 https://t.co/pHyaNuTvU6 to follow us on Facebook!

[Neurogene Inc. @NeurogeneInc]

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.

[Steve Grossman @stevenjgrossman]

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

[Richard Shelby @SenShelby]

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. https://t.co/glyQ2JqtKi

[Steve Grossman @stevenjgrossman]

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
https://t.co/m2hPfbUEjm

[Steve Grossman @stevenjgrossman]

Neurogene expands NO-COST genetic testing for children with unprovoked seizure age 5 years to under 8 years & joins Behind The Seizure https://t.co/WUUUPDvnv2 to improve diagnosis for patients with #raredisease Batten disease. #CLN5 #CLN8 #Battendisease
https://t.co/AaQA6MH5Hl

[Steve Grossman @stevenjgrossman]

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.
https://t.co/sfrH9vsNT8
#raredisease #viralvectors #CDMO

[Steve Grossman @stevenjgrossman]

Biotech leaders discussing #raredisease #genetherapy - "Expanding AAV Manufacturing" @NCATs_NIH.
Best practices, challenges, opportunities, logistics, analytics
Check out agenda & speakers and video available later https://t.co/pX9ATChoFA
#precisionmedicine #aav #celltherapy

[ADCY5.org Community @adcy5]

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

[ADCY5.org Community @adcy5]

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 https://t.co/wxmOVOseLy #EMR #EHR #HealthIT #DigitalHealth