Florie

383 posts

Florie

@flrbrl

Scientist at Arbor Bio helping to bring genomic medicines to the clinic #GeneTherapy #GeneEditing | Opinions shared are my own

Boston, MA Katılım Mayıs 2016

417 Takip Edilen448 Takipçiler

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I AM ALS@iamalsorg·25 Nis

Breaking news: The FDA has approved Tofersen for SOD1 ALS! @US_FDA thank you for living up to your promise of regulatory flexibility for ALS. ALS community: YOU made this happen with your relentless advocacy. Remember that. Stay tuned for updates on access to this treatment!

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Ammar Al-Chalabi@AmmarAlChalabi·4 Mar

30 years ago today the first cause of #ALS #MND was found by Prof Robert H Brown Jr and his team. Now we have a genetic therapy, Tofersen, that looks to have a dramatic effect in the 2% carrying this gene variant. A small step that shows ALS can be defeated with the right tools.

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Brian Wallach@bsw5020·14 Oca

I cried multiple times as I read @samstein’s truly beautiful article about me. I have been very public about my fight but for the first time I felt naked before the world. I used to fear this moment, but the poetry of Sam’s words made me thankful for it. politico.com/news/magazine/…

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Dr. Nicole Paulk@Nicole_Paulk·17 Kas

Friends in #Neuro #RareDisease #GeneTherapy --> new funding just dropped 👇

ASGCT@ASGCTherapy

Funding opportunities from the Ultra-rare Gene-based Therapy (URGenT) program (@NINDSnews) open Nov. 22. The program will provide funding and resources to advance #genetherapies for ultra-rare neurological diseases. Learn more and apply. bit.ly/3kI0ATr

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Florie retweetledi

Alpha-1 Foundation@AlphaFriend·8 Kas

Did you know that about 19 million people in the U.S. have one normal and one defective alpha-1 gene? For more Alpha-1 Fast Facts, please visit: alpha1.org/awareness #Alpha1Awareness

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Alpha-1 Foundation@AlphaFriend·3 Kas

The Alpha-1 Virtual Walk #A1FVW21 is all about meeting the Alpha-1 community where they are on their personal Alpha-1 journey. With over 75 registered teams from across the country, this year’s walk is gearing up to be the largest one yet! Read more here: alpha1.org/grab-your-snea…

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Alpha-1 Foundation@AlphaFriend·13 Eki

Have you registered for the Virtual Walk? Whether it’s around your dining room table, neighborhood, or local park - get up from your chair and get moving for Alpha-1 #A1FVW21! Sign up today: give.alpha1.org/site/TR?fr_id=…

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Endpoints News@endpts·2 Haz

Known as the ALS Functional Rating Scale, later revised in 1999, this scale has become one of the primary methods biopharmas have used to try to develop ALS drugs over the last 25 to 30 years. But some say it may be time for a more modern version. endpts.com/a-simple-patie…

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Florie@flrbrl·21 Nis

Thrilled to share the good news - our first safe to proceed @ApicBio! Hoping #GeneTherapy can make a difference for #ALS patients

Apic Bio@ApicBio

We are excited to announce @FDA clearance of our IND application for APB-102, our lead #genetherapy candidate. Read our announcement: bit.ly/3sCov7P

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Florie@flrbrl·28 Şub

#RareDiseaseDay

Rare Disease Day@rarediseaseday

Today is #RareDiseaseDay! There are over 6⃣0⃣0⃣0⃣ rare diseases 🌏 300 million people live with a rare disease accross the world Rare diseases currently affect 3.5% - 5.9% of the worldwide population👨‍👩‍👧‍👦 Learn more about what is a #raredisease 👉rarediseaseday.org/article/what-i…

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Florie@flrbrl·28 Şub

#RareDiseaseDay

Alpha-1 Foundation@AlphaFriend

#RareDiseaseDay at NIH will be held virtually on Monday, March 1, 2021, from 10:30 a.m. to 5:30 p.m. EST. This year’s event will include panel discussions, featuring our Clinical Director of the Alpha-1 Foundation Robert Sandhaus! To view the agenda, visit events-support.com/Documents/RDD_…

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Florie@flrbrl·28 Şub

#RareDiseaseDay

Rare Disease Day@rarediseaseday

📢 NEW! The official #RareDiseaseDay video is out NOW 🎉Watch the 6 real life stories and be inspired to join the 2021 campaign. 👥 Share this post with your followers using this hashtag #RareDiseaseDay

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Alpha-1 Foundation@AlphaFriend·10 Ara

Join us this Saturday, December 12, 2020 for the Virtual 20th Gordon L. Snider Critical Issues Workshop entitled "The Promise of Gene-based Interventions in Alpha-1 Antitrypsin Deficiency". Learn more about the scientific work being conducted towards finding a cure for Alpha-1!

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Antonio Regalado@antonioregalado·9 Ara

wicked big increases in viral RNA in Massachusetts sewage. (1/3)

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Florie@flrbrl·30 Kas

Ready for the #NCATSgenetherapy workshop

NCATS@ncats_nih_gov

Today, NCATS and @FDACBER are co-hosting the Virtual Workshop on Systemic Immunogenicity Considerations for AAV-mediated Gene Therapy. Register now to join us for the #NCATSGeneTherapy meeting at 9:30 a.m. EST: go.usa.gov/xfeQJ

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