rare diseases

Biotech Adam Feuerstein

Bluebird Bio’s gene therapy for blood disorders yields some impressive results ― but also raises questions

In the Lab Sharon Begley

In a medical first, ‘long read’ DNA sequencing solves a diagnostic mystery

Pharmalot Ed Silverman

Pharma seeks orphan designation for more drugs than ever before

Biotech Meghana Keshavan

Q&A: How to mimic a brand-name drug — with one key substitution

Politics Associated Press

Britain OKs making babies from DNA of three people in some cases

First Opinion Suzanne Koven

Why the election got me thinking about my patient’s painful struggle

Pharmalot Ed Silverman

Obama extends controversial program for rare pediatric drugs

Health Shayla Love

Is every disease a rare disease?

Pharmalot Ed Silverman

Congress tries to fix a drug voucher program, but critics say it’s not enough

In the Lab Karen Weintraub

Two newborn deaths in the same family set off a race to solve a medical mystery

Pharmalot Ed Silverman

Pharmalot, Pharmalittle: Valeant doubled price on assisted-suicide drug

Pharmalot Ed Silverman

FDA wants to nix voucher program for rare pediatric disease drugs

First Opinion Christopher Anselmo

How a blood test changed my life and helped me become a rare disease advocate

Health Andrew Joseph

Breaking down what the pope’s nod to birth control means for public health

Pharmalot Ed Silverman

Canadian provinces close the door on Alexion over price of rare disease drug

Pharmalot Ed Silverman

FDA designated a record number of orphan drugs last year

In the Lab Elie Dolgin

Beyond three-parent babies: New drugs offer hope for mitochondrial disease

Politics Ike Swetlitz

FDA urged to approve ‘three-parent embryos,’ a new frontier in reproduction

Pharmalot Ed Silverman

Why the FDA shouldn’t bow to parental pressure over Duchenne drug

In the Lab Karen Weintraub

A rare disease killed her mother. Can this scientist save herself?

Business Dylan Scott

This biotech company is trying to make a rare disease drug cheaper. Will it work?

In the Lab Rebecca Robbins

Big hopes for gene therapy dogged by uneven clinical results

Business Andrew Joseph

Drug developers fund patient registries to collect data on rare diseases

Pharmalot Ed Silverman

Bill creating more orphan drugs would cost taxpayers and patients

Health Melissa Bailey

No longer invisible: The faces of rare disease

Pharmalot Ed Silverman

Law for rare disease drugs needs revamping, researchers say

Business Helen Branswell

How a system meant to develop drugs for rare diseases broke down

Health Melissa Bailey

Boy’s mystery illness puts team of global scientists on the case

In the Lab Helen Branswell

The strange case of the man infected with tapeworm cancer