Lianna Orlando

VP of Research @CureDuchenne, Partner at CureDuchenne Ventures. #Duchenne #Neuroscience, #Neuromuscular, #RareDisease #VenturePhilanthrophy

[Lianna Orlando @LOrlandoScience]

Do you love a beautiful story of community, friendship, love, and the resilience of the human spirit, told via an award-winning documentary?

[CureDuchenne @CureDuchenne]

🎥 Watch The Remarkable Life of #Ibelin on #Netflix, the story of Mats Steen, a young man with Duchenne who found community in #worldofwarcraft 🙏 Thanks to the Steen family & @Blizzard_Ent for supporting #CureDuchenne with the #RevenPack fundraiser! 💙

https://t.co/bZJcl4WBk8

[World of Warcraft @Warcraft]

Adopt Reven & Reven’s Comfy Carrier to support CureDuchenne today!

Learn more: https://t.co/5lAsDVdRSU

[CureDuchenne @CureDuchenne]

JOIN THE WEBINAR: #FDA Decision on #Elevidys Explained
Date & TimeJun 25, 2024 3:00 PM in Pacific Time / 6:00 PM in Eastern Time: REG: https://t.co/UGM70pE7Jj

[Lianna Orlando @LOrlandoScience]

It's been a pleasure working w/ @MDAorg & @ParentProjectMD, in support of a study from @barrybyrne1 testing if a FDA-approved drug from @argenxglobal can lower pre-existing antibodies to AAV & enable more individuals with #Duchenne to have access to AAV-delivered #genetherapies

[CureDuchenne @CureDuchenne]

We're thrilled to work together to address one of the most pressing challenges in Duchenne: pre-existing antibodies to AAV, limiting who can be treated and re-dosed.
@MDAorg @ParentProjectMD
https://t.co/1s7uAiYNpE

[CureDuchenne @CureDuchenne]

We join the #BGTC in observing #RareDiseaseDay 2023 – together, we will accelerate and streamline therapies for rare diseases.

[CureDuchenne @CureDuchenne]

CureDuchenne Ventures is pleased to share the news of our latest investment.

hC Bioscience, Inc.’s experimental therapy aims to correct premature stop codon mutations in Duchenne to restore expression of full-length dystrophin.
https://t.co/RuoLKh13Gn

[Lianna Orlando @LOrlandoScience]

I am proud to represent CureDuchenne in this collaborative effort, so that more rare diseases can benefit from safe and effective gene therapies

[CureDuchenne @CureDuchenne]

Did you know that there are more than 10,000 rare diseases that we know of? CureDuchenne is proud to be a partner in the Bespoke Gene Therapy Consortium to create a gene therapy protocol that can be applied to many of these diseases.

[CureDuchenne @CureDuchenne]

CureDuchenne provided initial seed funding to Myosana in 2020. We welcome new investors as Myosana closes its latest round of funding to support further development of their non-viral gene therapy technology.
https://t.co/qYMmZhMDVb

[Lianna Orlando @LOrlandoScience]

I’m proud to represent @CureDuchenne in this important #GeneTherapy effort. A lot of work completed in one year, and many exciting developments ahead!

[Foundation for the National Institutes of Health @FNIH_Org]

AMP® BGTC is celebrating its one-year anniversary! Learn more about this groundbreaking gene therapy effort, what the consortium has accomplished in year one, and what’s in store for year two: https://t.co/97TyQbDqac

[Lianna Orlando @LOrlandoScience]

This milestone in #Duchenne muscular dystrophy has been decades in the making. Congrats to @Sarepta $SRPT and thank you to everyone who has worked on #GeneTherapy over the years to help get us here!

[Sarepta Therapeutics @Sarepta]

NEWS: SRPT today announced it has completed the BLA submission seeking accelerated approval for SRP-9001 to treat ambulant individuals with #Duchenne. SRP-9001 is an investigational gene therapy being developed in partnership with Roche. Read the release: https://t.co/l4cn5lmnMk

[Edgewise Therapeutics @EdgewiseTx]

We are growing our cardiovascular team and looking for roles at all levels! If you’re passionate about CV research and development, reach out to [email protected] to learn more. https://t.co/R0Wk5GjDg2

[PepGen @PepGenTx]

Today we announced exciting #DMD results – PGN-EDO51 achieved the highest level of oligonucleotide delivery and exon 51 skipping following a single dose in humans. We will host a corresponding conference call and a live webcast at 8:00 a.m. ET. Read more: https://t.co/bFOMwv28bd

[Lianna Orlando @LOrlandoScience]

News that Sarepta intends to submit of accelerated approval for #DMD #Duchenne #GeneTherapy

[Sarepta Therapeutics @Sarepta]

Our latest Community Bulletin provides an update on our intention to seek an accelerated approval from the U.S. FDA for SRP-9001 to treat individuals with Duchenne muscular dystrophy. Read the bulletin: https://t.co/NMo0tqJnNE

[Lianna Orlando @LOrlandoScience]

@Matt_Muscle_Guy I won’t be there this time, so looking forward to your updates!

[PepGen @PepGenTx]

Today we are celebrating PepGen’s #IPO and our first trade on the @Nasdaq as $PEPG! Thanks to our team and investors for making this day possible, and to the rare muscle disease community for supporting our science & mission. #PepGenPioneers

[Lianna Orlando @LOrlandoScience]

Pfizer gets go-ahead to re-start Phase 3 #GeneTherapy trial in #Duchenne muscular dystrophy. US sites opening soon. $PFE https://t.co/Lf85OoyX8e

[Lianna Orlando @LOrlandoScience]

@WashUNeurology @LabWeihl Congratulations Chris!

[Lianna Orlando @LOrlandoScience]

@4jakes_sake Happy Birthday and Congratulations!!

[CureDuchenne @CureDuchenne]

@Sarepta announced promising results from Part 2 of Study 102, an ongoing DMD clinical trial of investigational gene therapy SRP-9001. https://t.co/uUbMdx33KJ