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- PMLiVE

Raptor Pharmaceutical

- PMLiVE

Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions

Join us live at 1.00pm (GMT)/2.00pm (CET)/8.00am (EST) on Thursday 29th February, to explore rare disease market access challenges and learn more about these high potential markets.

Mtech Access

- PMLiVE

Horizon looks bright as FDA panel backs eye drug teprotumumab

First alternative to surgery for proptosis

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- PMLiVE

Horizon sunny as thyroid eye disease drug hits the mark

New treatment helps lessen eye bulging

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

Rare diseases and orphan drugs: Our experience and expertise

We have designed a complimentary infographic outlining the market opportunity as well as our experience and expertise in the growing area of rare diseases and orphan drugs, which includes links to...

Research Partnership

Malthus’ orphans

Firms focusing on small, high-profit niches risk falling into a 200-year-old trap

- PMLiVE

EMA hands UniQure orphan status first in Huntington’s

The Dutch biotech expects to file the treatment for approval later this year

Roche Basel Switzerland

Roche claims its own CAR-T first with orphan drug Actemra

Comes as FDA approves the first CAR-T therapy from Novartis

- PMLiVE

FDA moves to tackle orphan designation backlog

Some 200 requests are awaiting review by the US regulator

Shire Basingstoke

Shire finally bags EU approval for rare disease drug Natpar

Becomes first licenced treatment for hypoparathyroidism in Europe

Blending medical and digital for effective rare disease campaigns

The fundamentals of building a collaborative medical and digital team

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