Articles & Podcasts

N=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth

Articles & Podcasts

UNC Fast-Tracks Personalized Treatment for Twins with Ultra Rare Genetic Disorder

Articles & Podcasts

Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases

Articles & Podcasts

Building a Bridge Between Rare Disease Patients and the Technologies that Could Help Them

Articles & Podcasts

Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests

Articles & Podcasts

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...

Articles & Podcasts

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...

Articles & Podcasts

This lifesaving treatment was designed for one. Could it be the future of medical care?

Articles & Podcasts

Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease

Articles & Podcasts

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...

Articles & Podcasts

The world’s first pathway for individually designed drugs

Articles & Podcasts

Collaboratives, Companies Mobilize to BuildMomentum for 'N-of-1' ASO Therapies

Articles & Podcasts

Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient

Articles & Podcasts

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...

Articles & Podcasts

Individualised medicines offer hope for children with a rare disease

Articles & Podcasts

Q&A: FDA's Peter Marks ready to encourage more accelerated approvals for rare diseases

Articles & Podcasts

Too many treatable diseases go unnoticed. This could change that.

Articles & Podcasts

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

Articles & Podcasts

The quest for the era of personalised medicine

Articles & Podcasts

N=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth

UNC Fast-Tracks Personalized Treatment for Twins with Ultra Rare Genetic Disorder

Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases

Building a Bridge Between Rare Disease Patients and the Technologies that Could Help Them

Can AI build custom ‘n-of-1’ drugs faster? Maybe not yet, new case study suggests

Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says

Four Keys To Advancing Antisense Oligonucleotides For Personalized Medicine

This lifesaving treatment was designed for one. Could it be the future of medical care?

Oxford-Harrington Rare Disease Centre named first global initiative for children with rare disease

Pilot launched to support children with rare conditions to access personalised therapies

The world’s first pathway for individually designed drugs

Collaboratives, Companies Mobilize to BuildMomentum for 'N-of-1' ASO Therapies

Early Rare Disease Diagnosis Could Save as Much as $500,000 per Patient

What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists

Individualised medicines offer hope for children with a rare disease

Q&A: FDA's Peter Marks ready to encourage more accelerated approvals for rare diseases

Too many treatable diseases go unnoticed. This could change that.

‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

The quest for the era of personalised medicine

A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments

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