Tim Yu

@timyu

Neurology, Genetics, Genomic Medicine. Tweets my own unless one of our girls has broken into my account again in which case sorry for all the Pokémon pics

Vrijeme pridruživanja: srpanj 2007.

Tweetovi

Blokirali ste korisnika/cu @timyu

Jeste li sigurni da želite vidjeti te tweetove? Time nećete deblokirati korisnika/cu @timyu

  1. Prikvačeni tweet
    10. lis 2019.

    1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at . It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA ().

    Prikaži ovu nit
    Poništi
  2. proslijedio/la je Tweet
    4. velj

    If you’re interested in then this week’s ⁦⁩ pod is a must-listen; huge thanks to ⁦⁦⁩ of ⁦⁩ for joining us to discuss milasen for his patient with Batten Disease! ⁦

    Poništi
  3. proslijedio/la je Tweet
    9. sij

    In the "proud tradition of activated patients driving science," the remarkable and updated story of Sonia Vallabh and including a healthy baby via PGD, their PhDs and labs, great ASO progress, + driving a big Δ in approach

    Poništi
  4. 8. sij

    The scientific foundation for our milasen work was Spinraza, birthed by Adrian Krainer & Frank Bennett. Just 1 of the reasons I’m grateful to & for this launch, supporting more individualized txs for ultrarare disease.

    Poništi
  5. 27. pro 2019.

    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.

    Poništi
  6. 3. pro 2019.

    This I'm proud of Tori Suslovitch, our genetic counselor who's running the Boston Marathon to raise money to support and our work to help children with rare neurogenic disorders around the globe:

    Poništi
  7. proslijedio/la je Tweet
    28. stu 2019.

    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!

    Poništi
  8. 28. stu 2019.
    Poništi
  9. proslijedio/la je Tweet

    Mila Makovec was born with a rare condition. But a drug made just for her has changed the course of her disease. What it means for the future of on my blog.

    Poništi
  10. proslijedio/la je Tweet
    14. lis 2019.

    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.

    Poništi
  11. 14. lis 2019.

    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.

    Poništi
  12. proslijedio/la je Tweet
    13. lis 2019.

    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.

    Poništi
  13. 13. lis 2019.

    Congratulations Tojo, Aubrie, Diana and Tori, representing the Yu lab at the ! Way to go!

    Poništi
  14. 10. lis 2019.

    [4/3: Now, back to work :)]

    Prikaži ovu nit
    Poništi
  15. 10. lis 2019.

    3/3 Last, grateful for the trust and partnership of our patient’s family and Mila’s Miracle Foundation (). This serves as a public start of important conversations about carefully considered scientific, ethical, and regulatory frameworks for individualized medicine.

    Prikaži ovu nit
    Poništi
  16. 10. lis 2019.

    2/3 Congrats to lead authors & April Hu. Proud of the pioneering support from colleagues at as well as , , , , , , BrammerBio, , and many more.

    Prikaži ovu nit
    Poništi
  17. proslijedio/la je Tweet
    24. srp 2019.

    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

    Poništi
  18. 8. srp 2019.
    Poništi
  19. proslijedio/la je Tweet
    24. lip 2019.
    Poništi
  20. 17. lip 2019.

    Out today in : 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!

    Poništi

Čini se da učitavanje traje već neko vrijeme.

Twitter je možda preopterećen ili ima kratkotrajnih poteškoća u radu. Pokušajte ponovno ili potražite dodatne informacije u odjeljku Status Twittera.

    Možda bi vam se svidjelo i ovo:

    ·