rare disease

First Opinion By William S. Smith

The Biden administration needs to look beyond ICER for evaluating drug therapies

In the Lab By Sharon Begley

In a stem cell experiment that seemingly failed, scientists see a possible way to prevent a devastating disease

Health By Anna Goshua

‘Frustrated and panicking’: For some rare disease patients, shortages of protective gear pose a continued threat

Biotech By Damian Garde

Ovid’s treatment for a rare, untreatable disease fails in a key trial

Pharmalot By Ed Silverman

House passes a bill to close a loophole in a law that gives drug makers ‘unfair’ monopolies

By Damian Garde, Meg Tirrell, and Adam Feuerstein

Listen: Bracing for a pandemic winter, Biogen’s fortune at the FDA, and remembering a remarkable 12-year-old

First Opinion By Wolfram Nothaft, Gregory Moore, and Yann Le Cam

Using telehealth to revolutionize the speed of making rare disease diagnoses

Biotech By Elizabeth Cooney

Wary hemophilia patients say they’re willing to wait longer for a safe gene therapy

Biotech By Adam Feuerstein and Damian Garde

Stunning FDA rejection signals potential new hurdles for gene therapies

Pharmalot By Ed Silverman

A long-awaited report on orphan drugs in Europe suggests incentives to pharma need change

In the Lab By Sharon Begley

Gene-editing discovery could point the way toward a ‘holy grail’: cures for mitochondrial diseases

First Opinion By Michelle McMurry-Heath

A pandemic, a funeral, and a chance to help heal the world

First Opinion By Christopher Anselmo

I’ve been cared for by dozens of clinicians in nearly 20 years. I’m in awe of their work on Covid-19

Pharmalot By Ed Silverman

FDA closes review of Novartis data integrity scandal without punishing the drug maker

Biotech By Damian Garde

To quell unpredictable allergic reactions, an experimental drug takes aim at a genetic cause, not symptoms

Pharmalot By Ed Silverman

Oh, Canada! Vertex battles another country over access to cystic fibrosis treatments

First Opinion By Ethan J. Weiss

Should ‘broken’ genes be fixed? My daughter changed the way I think about that question

Pharmalot By Ed Silverman

Pharma execs criticize a drug maker over pricing and claim orphan drug law is being misused

Off the Charts By Jennifer Adaeze Okwerekwu

As medical professionals, my husband and I know the Nigerian travel ban is cruel. Now the pain is personal

Health By Andrew Joseph and Ed Silverman

A lottery like no other offers up a cutting-edge medicine — with lives on the line

Adam's Take By Adam Feuerstein

If biotech had a Mount Rushmore, whose heads would you chisel in the granite?

Health By Rebecca Robbins

Chan Zuckerberg Initiative to fund 30 patient groups, aiming to build a model for tackling rare diseases

Biotech By Matthew Herper, Adam Feuerstein, and Damian Garde

What does the latest twist in the Sarepta saga mean for the future of the FDA? We discuss

Biotech By Adam Feuerstein

On the cusp of FDA approval for its NASH drug, Intercept’s CEO talks payers, pruritis, and price

Biotech By Jonathan Saltzman — Boston Globe

Blueprint gains approval for its first drug, a treatment for a rare gastrointestinal cancer