rare diseases

Off the Charts By Jennifer Adaeze Okwerekwu

When the hoofbeats really are a zebra’s, a patient community helps me navigate a new rare-disease reality

Biotech By Jonathan Saltzman — Boston Globe

College student with disease that causes premature aging is heartened by medical advances

Pharmalot By Ed Silverman

Pharmalittle: FDA places hold on Novartis trial for gene therapy; FDA floats plan for combating shortages

Pharmalot By Ed Silverman

Consumer groups urge Italian and Belgian authorities to investigate Biogen over Spinraza pricing

Biotech By Adam Feuerstein

Gene therapies are a hard sell. Bluebird hopes to do better in Europe

Pharmalot By Ed Silverman

Laura Jacobus hopes to provide her newly approved drug at ‘little or no’ cost to patients

Pharmalot By Ed Silverman

Pharmalittle: Pfizer prices new rare disease drug at $225,000; Novartis pushes gene therapy

Pharmalot By Ed Silverman

Pharmalittle: Appaloosa says Allergan moves are not enough; FDA rejects Sanofi diabetes pill

Pharmalot By Ed Silverman

Pharmalittle: Pfizer pays $636 million for rights to gene therapies; Novartis denies bribing Greek officials

Pharmalot By Ed Silverman

Catalyst says its pricey rare disease drug is doing ‘better than expected’ despite patient anger

Pharmalot By Ed Silverman

Sanders asks FDA to allow sales of older versions of $375,000 rare-disease drug

Biotech By Damian Garde

Amicus’ rare disease drug, once indefinitely delayed, wins FDA approval

Biotech By Jonathan Saltzman — Boston Globe

Trials for potential ALS treatment using stem cells spark cautious hope

Pharmalot By Ed Silverman

Parents accuse companies of stealing medical data from their very sick children

Pharmalot By Ed Silverman

Rare diseases and trade secrets: Shire and Ultragenyx fight over lists of doctors

Biotech By Adam Feuerstein

With tweaks to gene therapy, Bluebird Bio makes progress in sickle cell

Business By Adam Feuerstein

The new Biotech Scorecard: 10 make-or-break industry events for the fourth quarter

Biotech By Adam Feuerstein

FDA rips into PTC’s Duchenne muscular dystrophy drug as advisory panel looms

Politics By Damian Garde

Is the FDA telegraphing a rejection for PTC Therapeutics’ DMD drug?

Biotech By Damian Garde

Key partner cuts ties with brash biotech startup Moderna, raising big questions about its pipeline

Biotech By Adam Feuerstein

FDA approves first new sickle cell drug in 20 years

Biotech By Adam Feuerstein

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

In the Lab By Sharon Begley

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

Pharmalot By Ed Silverman

Pharma seeks orphan designation for more drugs than ever before

Biotech By Meghana Keshavan

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