1/2 Just out: "Timely Development of a Mutation-Specific Drug", with an accompanying editorial, on the front page of NEJM. Congrats to the lead author Jinkuk Kim (a postdoc in the lab), who worked with our collaborator Tim Yu on this amazing story.

Many more patients with unique mutations can benefit from individualized oligo therapeutics!Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease | NEJM nejm.org/doi/full/10.10…

Congrats Tim Yu, Jinkuk Kim, Mila, her family, and the many others who took part in making this incredible journey to milasen happen! @Manton is proud to work with this amazing team Boston Children's #raredisease

1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at nejm.org/doi/full/10.10…. It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (nejm.org/doi/full/10.10…).

Truly remarkable work by Tim Yu and team - and a glimpse of the future of N-of-1 therapies for ultra-rare diseases.

In the era of individualized medicine, the 1st drug designed for a single patient. Named after her, helping to treat an incurable condition. nejm.org/doi/full/10.10… NEJM by Tim Yu and colleagues Boston Children's nytimes.com/2019/10/09/hea… statnews.com/2019/10/09/fda… by Meghana Keshavan

This is amazing. In case you were looking for evidence that we are living in the most exciting period of science & medicine in history... Tim Yu Jinkuk Kim Boston Children's #personalizedmedicine #PrecisionMedicine nejm.org/doi/full/10.10…

I’m late to this N=1 oligo Rx paper — but I heard lead author Jinkuk Kim speak a few weeks ago, and he was 💯👍⭐️, & sr author Tim Yu is a great guy. For a more accessible read, here’s Gina Kolata’s A+ NYT piece: nytimes.com/2019/10/09/hea… nejm.org/doi/full/10.10…

Congrats to Jinkuk Kim from Tim Yu's lab on this amazing story of treating a patient's genetic disease caused by a transposon insertion and defective RNA splicing. nejm.org/doi/full/10.10…

N of 1 study of patient-customized oligonucleotide therapy adopting mRNA splice-switching strategy in a patient with rare genetic disease (Batten’s disease). Hope to see this level of individualization realized for cancer treatment. nejm.org/doi/full/10.10…

Lessons Learned by Eric Schmidt transcribed by Abheek Anand (recommended by troy simpson 📈) gist.github.com/xcud/26b6e3135…

That "5 minutes chat" with a developer. ❤️ if you agree.

It's easier to use Sci-Hub to access papers than it is to use publisher websites. Even if you have inst. access! Just type title, get PDF.

The world’s 10 biggest economies in 2017 wef.ch/2tiRwbt #economics