Rare Diseases

In the Lab By Elizabeth Cooney

‘It’s the disparity in who gets affected’: A sickle cell doctor tracks dollars and drug development

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

Meghan Waldron is a freshman at Emerson College with progeria, one of the world's rarest diseases.

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

Pharmalot By Ed Silverman

Catalyst sues FDA over rivals’ drug approval, warning it undermines incentives for rare disease meds

Biotech By Adam Feuerstein

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

Biotech By Damian Garde and Adam Feuerstein

Your guide to the mounting competition among new SMA drugs

Pharmalot By Ed Silverman

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

Pharmalot By Ed Silverman

In a crafty move, FDA may have found a way to dampen controversy over a $375,000 rare-disease drug

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

Health By Meghana Keshavan

We may soon have our first $1 million drug. Who will pay for it? And how?

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

Most Read

Jacobus prices its rare disease drug at half of what Catalyst charges, but will doctors prescribe it?

How much would you pay for the miracle of gene therapy?

To save a young woman besieged by superbugs, scientists hunt a killer virus

FDA approves first new sickle cell drug in 20 years

Searching for a diagnosis: a network of doctors tries to solve medical mysteries

My son had a devastating, mysterious illness. The conversation about palliative care knocked me flat

A quest to explain the whooshing in her head

A family-run drug maker faces tough choices to stay afloat in the Shkreli era

All Coverage

‘It’s the disparity in who gets affected’: A sickle cell doctor tracks dollars and drug development

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

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

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

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

Catalyst sues FDA over rivals’ drug approval, warning it undermines incentives for rare disease meds

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

Your guide to the mounting competition among new SMA drugs

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

In a crafty move, FDA may have found a way to dampen controversy over a $375,000 rare-disease drug

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

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

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

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

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

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

Trials for potential ALS treatment using stem cells spark cautious hope

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

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

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

We may soon have our first $1 million drug. Who will pay for it? And how?

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

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

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

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

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rare diseases

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