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Prikvačeni tweet
1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at https://www.nejm.org/doi/full/10.1056/NEJMoa1813279 …. It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (https://www.nejm.org/doi/full/10.1056/NEJMe1911295 …).
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Tim Yu proslijedio/la je Tweet
If you’re interested in
#precisionmedicine then this week’s @GreenJournal pod is a must-listen; huge thanks to @timyu of @BostonChildrens for joining us to discuss milasen for his patient with Batten Disease! @AANMemberhttps://podcasts.apple.com/us/podcast/neurology-podcast/id266288159?i=1000464509889 …Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Tim Yu proslijedio/la je Tweet
In the "proud tradition of activated patients driving science," the remarkable and updated story of Sonia Vallabh and
@cureffi@NEJM https://www.nejm.org/doi/full/10.1056/NEJMp1909471?query=featured_home … including a healthy baby via PGD, their PhDs and labs, great ASO progress, + driving a big Δ in approach@broadinstitutepic.twitter.com/BgspCeYTOo
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The scientific foundation for our milasen work was Spinraza, birthed by Adrian Krainer & Frank Bennett. Just 1 of the reasons I’m grateful to
@DrStanleyCrooke@ionispharma &@biogen for this launch, supporting more individualized txs for ultrarare disease.http://bit.ly/cennloremHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
This. One of the most important lessons for trainees (and doubly important to convey to families working with scientists) is the fundamental uphill nature of science. Experiments by their nature fail. The trick is rigging them to learn a little each time.https://twitter.com/davidrliu/status/1210577205119651843 …
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This
#GivingTuesday I'm proud of Tori Suslovitch, our genetic counselor who's running the Boston Marathon to raise money to support@BostonChildrens and our work to help children with rare neurogenic disorders around the globe:http://fundraise.childrenshospital.org/goto/victoriaHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Tim Yu proslijedio/la je Tweet
@OTSociety has formed a Rare Disease Task Force to work with other stakeholders in facilitating the development of N-of-1+ oligonucleotide therapeutics - lots going on now!@stopbatten@timyuhttps://twitter.com/OTSociety/status/1200073149276151808 …
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Tim Yu proslijedio/la je Tweet
Mila Makovec was born with a rare
#genetic condition. But a drug made just for her has changed the course of her disease. What it means for the future of#precisionmedicine on my blog.#NIHhttp://bit.ly/2JjmoC6Hvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Tim Yu proslijedio/la je Tweet
Opinion: A more centralized, standardized, and predictable FDA-wide approach to best practices for new drug-development policy will encourage developers to use more cutting edge, efficient, and swifter approaches to bringing lifesaving therapies to market.https://buff.ly/2B9Dgqk
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Nancy Fliesler and her team w/milasen’s backstory -- how an offer to help a family w/a diagnosis led to an unexpected journey, w/many collaborators along the way. http://www.childrenshospital.org/research/batten
@BCH_Innovation@BostonChildrens@nansonahamHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Tim Yu proslijedio/la je Tweet
Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, spoke with STAT about the promise of treatments for patients with ultra-rare diseases — and how they might be developed on a larger scale.https://buff.ly/2BaSMlC
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Congratulations Tojo, Aubrie, Diana and Tori, representing the Yu lab at the
#BAAHalfMarathon! Way to go!pic.twitter.com/u9mu5St4LC
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3/3 Last, grateful for the trust and partnership of our patient’s family and Mila’s Miracle Foundation (
@stopbatten). This serves as a public start of important conversations about carefully considered scientific, ethical, and regulatory frameworks for individualized medicine.Prikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
2/3 Congrats to lead authors
@jinkukkim & April Hu. Proud of the pioneering support from colleagues at@BostonChildrens as well as@ChildrensColo,@NUFeinbergMed,@UR_Med,@harvardmed,@CRiverLabs,@TriLinkBioTech, BrammerBio,@US_FDA, and many more.Prikaži ovu nitHvala. Twitter će to iskoristiti za poboljšanje vaše vremenske crte. PoništiPoništi -
Tim Yu proslijedio/la je Tweet
The potential of antisense oligos to treat rare disease is just beginning to be harnessed. An accelerator could help promote this, building on the recent successes in the field
@OTSociety@timyu@stopbattenhttps://twitter.com/rohanseth/status/1154105234462871552 …
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Such tough news to have to share. Hugh and Chris, our thoughts are with you. https://www.rgj.com/story/news/2019/07/05/addi-and-cassi-hempel-neimann-pick-disease-type-c-have-died-reno/1659215001/ …https://twitter.com/AmyDMarcus/status/1147232454832529408 …
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Tim Yu proslijedio/la je Tweet
Rare recessive mutations pry open new windows on
#autism - via@timyu, Ryan Doan & colleagues@NatureGenet: http://discoveries.childrenshospital.org/recessive-autism-mutations/ …#genetics#ASDpic.twitter.com/Lgy3AhtGnw
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Out today in
@NatureGenet: our group's work w/the Autism Sequencing Consortium examining recessive mutations, extending the female protective effect, and uncovering new evidence linking serotonin & ASD. Congrats to all involved, esp 1st author Ryan Doan!https://twitter.com/NatureGenet/status/1140642802163957760 …
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