They Created a Drug for Susannah. What About Millions of Other Patients?
By Erika Check Hayden (New York Times) Photographs by Brittainy Newman Scientists have made rapid progress in customizing drugs for...
top of page
Search
Hopes for Antisense Oligonucleotides Remain Strong Despite Severe Adverse Events in Trials
By Dan Hurley (https://journals.lww.com/) Two children who were administered an antisense oligonucleotide (ASO) for a severe genetic...
38 views
We Can Cure Disease by Editing a Person’s DNA. Why Aren’t We?
Fyodor Urnov (NY Times) December 9, 2022 The parents of a 2-year-old girl write that their daughter “could die within the next year”...
12 views
Gene Treatment for Rare Epilepsy Causes Brain Side Effect in 2 Children
Erika Check Hayden (NY Times) October 26, 2022 Weeks after Valeria Schenkel took an experimental drug named after her, the daily seizures...
22 views
From Mila to millions
"We took a look and we perform whole genome sequencing of Mila, her mother, her father, as well as her little brother. And we looked...
18 views
A mother, shaped by tragedy, embarks on a mission to advance custom medicines
Jared Whitlock (STAT News) Feb. 9, 2022 For weeks, Julia Vitarello avoided the room in her home. The fairy curtains she sewed. The...
15 views
If DNA is like software, can we just fix the code?
When you first meet her, you won’t be able to tell that Ipek Kuzu suffers from a rare genetic disease. The three-year-old plays happily...
7 views
Drug tailored to one girl with brain disease paves way for similar customized treatments
Jocelyn Kaiser (Science) October 9, 2019 After their apparent success treating a young girl with a drug tailored to counteract a genetic...
7 views
This girl’s dramatic story shows hyper-personalized medicine is possible—and costly
When six-year-old Mila Makovec was diagnosed with a devastating neurological disorder called Batten disease, the happy, active girl had ...
6 views
After a bespoke therapy rescues a young girl, the FDA considers advance of individualized treatments
Meghana Keshavan (STAT News) October 9, 2019 Back in 2016, a 6-year-old named Mila was diagnosed with Batten disease, a progressive and...
7 views
When ‘right to try’ isn’t enough: Congress wants a single ALS patient to get a therapy never tested
Nicholas Florko (STAT News) May 31, 2019 WASHINGTON — A family in Iowa believes the Food and Drug Administration will decide whether...
11 views
bottom of page