Rare Disease

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Sanofi claims FDA okay for drug to treat rare blood disorder...

Sanofi has won FDA approval for its sutimlimab for cold agglutinin disease (CAD) at the second time of asking, becoming the first approved therapy for the rare blood disorder in the US.

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DNA helix in a futuristic concept of the evolution of science and medicine.

NHS signs deal with Orchard for pricey Libmeldy gene therapy

NHS England has agreed a confidential deal with Orchard Therapeutics that will allow access to Libmeldy – a gene therapy for a rare childhood disease that is thought to be the most expensiv

Market Access

How does rare disease prevalence impact drug pricing in Engl...

In this second article of a three-part series exploring pricing in Europe, CRA’s Life Sciences Practice team discusses the role that disease rarity plays in reimbursement decisions among pa

News

group of chemical scientists working at the laboratory.

Pharming preps filing for rare disease drug leniolisib after...

Pharming has taken a step closer to getting a second product to market, after its rare disease therapy leniolisib met its objectives in a pivotal trial, setting up regulatory filings in the

News

After FDA setback, Ipsen gets first OK for rare disease drug...

After regulatory hold-ups in the US and Europe, Ipsen has claimed a confidence-boosting approval in Canada for palovarotene, a rare disease therapy acquired via its $1.3 billion takeover of

News

Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Sarepta says early filing for DMD gene therapy may be back o...

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file fo

Sanofi claims FDA okay for drug to treat rare blood disorder...

NHS signs deal with Orchard for pricey Libmeldy gene therapy

How does rare disease prevalence impact drug pricing in Engl...

Pharming preps filing for rare disease drug leniolisib after...

After FDA setback, Ipsen gets first OK for rare disease drug...

Sarepta says early filing for DMD gene therapy may be back o...

CEPI steps in to fund Moderna's pandemic flu jab

How to meet your physicians where they are online

Rare Disease

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