top of page
Search
N=1 Collaborative: FDA’s New Plausible Mechanism Pathway
November 19, 2025 By Julia Pian, MD Last Wednesday, the U.S. Food and Drug Administration announced the Plausible Mechanism Pathway, outlining in broad strokes a new regulatory strategy for supporting the responsible advancement of precision genetic therapies [1]. As an independent, cross-sector non-profit, the N=1 Collaborative welcomes this move, signaling the FDA's willingness to be a creative partner in developing models to unlock the potential of precisely targeted, pr
How a tailored ALS drug could help broaden rare disease therapies
Jared Whitlock ( Endpoints News ) June 18, 2025 In 2020, Jaci Hermstad died at age 26 from a rare and aggressive form of ALS. But her...
The new era of individualized medicine requires a ‘genetic surgery’ system
Julia Vitarello ( STAT ) May 22, 2025 In 2018, my daughter, Mila, became the first person in the world to receive a medicine designed for...
A Baby Receives the First Customized CRISPR Treatment
Alice Park ( TIME ) May 15, 2025 G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential...
'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis
Ryan Cross ( Endpoint News ) May 8, 2025 Fyodor Urnov had given a version of his speech dozens of times before. Yet as he leaned into the...
N=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth
OCT 3, 2024 BOSTON, October 3, 2024 ( Newswire.com ) - The N=1 Collaborative (N1C), a non-profit organization representing over 700...
UNC Fast-Tracks Personalized Treatment for Twins with Ultra Rare Genetic Disorder
UNC Health and UNC School of Medicine Newsroom September 25, 2024 In May 2022, Yael Shiloh-Malawsky, MD, a neurologist at the UNC School...
Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases
Gerry Smith ( Bloomberg ) September 5, 2024 As a wave of gene therapies with multimillion-dollar price tags hit the market, many...
Building a Bridge Between Rare Disease Patients and the Technologies that Could Help Them
Laura Cowen (Inside Precision Medicine) June 4, 2024 In recent years, whole genome sequencing has made it easier to diagnose rare genetic...
Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests
Jason Mast (STAT News) May 13, 2024 Two years ago, Yiwei She was searching desperately for someone to build a drug for her newborn son,...
Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says
Zachary Brennan (Endpoints News) February 27, 2027 The FDA will increasingly use accelerated approval for gene therapies aimed at rare...
Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine
Demaris Mills (Forbes) January 12, 2024 The 2023 Oligonucleotide Therapeutics Society meeting drew 800 attendees to Barcelona, a clear...
This lifesaving treatment was designed for one. Could it be the future of medical care?
Karen Weintraub (USA Today) November 26, 2023 Imagine you or your child gets a diagnosis so rare no one else on Earth is known to have...
Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease
University of Oxford November 24, 2023 In the autumn statement on Wednesday 22 November, the government announced its support for the...
Pilot launched to support children with rare conditions to access personalised therapies
Genomics England November 22, 2023 New programme announced to develop a pathway for children with rare conditions to access therapies...
The world’s first pathway for individually designed drugs
The Economist November 22, 2023 The headlines about the autumn statement from Jeremy Hunt, the chancellor, this week focused on tax and...
Collaboratives, Companies Mobilize to BuildMomentum for 'N-of-1' ASO Therapies
Jessica Kim Cohen (Precision Medicine Online) October 30, 2023 NEW YORK – A neurologist at Boston Children's Hospital in 2018 created...
Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient
September 15, 2023 New research by the EveryLife Foundation for Rare Diseases, in one of the first studies of healthcare utilization and...
What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists
October 12, 2023 I first saw Terry Horgan as a patient. I was filling in as the pediatric pulmonary specialist in the UMass-Duchenne...
Individualised medicines offer hope for children with a rare disease
Henry Phillips (Health Awareness) September 1, 2023 What started as a race against time for my daughter Mila, diagnosed at six with a...
bottom of page