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Pharmalot Elizabeth Cooney

Pharmalittle: Advisory panel narrowly votes down ALS drug; Vertex looks to advance experimental pain drug

In the Lab Megan Molteni

New CRISPR tools could fix diseases caused by large DNA rearrangements, scientists report

Pharmalot Ed Silverman

After repeated losses in the U.S., Catalyst wins a victory in Canada over a rare disease drug

Pharmalot Ed Silverman

Pharmalittle: White House signs deal for U.S.-made generics to fight Covid-19; Gilead expands remdesivir donations

In the Lab Elizabeth Cooney

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

Off the Charts Jennifer Adaeze Okwerekwu

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

Biotech Jonathan Saltzman — Boston Globe

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

Pharmalot Ed Silverman

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

All Coverage

Biotech Damian Garde and Adam Feuerstein

Your guide to the mounting competition among new SMA drugs

Pharmalot Ed Silverman

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

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Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Biotech Damian Garde

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

First Opinion Maria Kefalas

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

Biotech Jonathan Saltzman — Boston Globe

Trials for potential ALS treatment using stem cells spark cautious hope

Pharmalot Ed Silverman

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

Pharmalot Ed Silverman

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

Health Eric Boodman

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

Biotech Adam Feuerstein

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

Health Meghana Keshavan

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

Business Adam Feuerstein

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