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- PMLiVE

Orfadin

- 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

Sanofi and Sobi announce positive phase 3 trial results for severe haemophilia A

Results from the study showed once-weekly efanesoctocog alfa doses provided ‘clinically meaningful’ bleed protection for patients with severe haemophilia A

- PMLiVE

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

- 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

ABPI London offices

UK pharma slams new drug evaluation plans

ABPI and BIA oppose "arbitrary" proposal to impose £100,000 QALY limit on rare disease therapies

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