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PTC Therapeutics' chief executive Matthew Klein.

Germany will be first launch for PTC's phenylketonuria drug

PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.

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Dupixent is first FDA-approved drug for rare skin disease

The long list of therapeutic uses for Sanofi and Regeneron's Dupixent has been lengthened by an FDA approval for rare skin disease bullous pemphigoid.

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Biogen bids to extend use of Skyclarys to younger patients

Biogen has started a new phase 3 trial that aims to extend the use of its Friedreich's ataxia drug Skyclarys to patients as young as two.

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Guidance creates access gap in UK for GPP drug Spevigo

People in England and Wales with severe skin disease GPP will soon be able to access a new therapy currently denied to patients in Scotland.

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FDA green lights CSL's once-monthly HAE drug Andembry

CSL has won FDA approval for Andembry, its once-monthly subcutaneous drug to prevent hereditary angioedema (HAE) attacks.

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Sanofi draws up $9.1bn plan to buy Blueprint Meds

Sanofi has bolstered its rare disease business with an agreement to buy Blueprint Medicines and blockbuster-in-waiting Ayvakit for ASM.

Germany will be first launch for PTC's phenylketonuria drug

Dupixent is first FDA-approved drug for rare skin disease

Biogen bids to extend use of Skyclarys to younger patients

Guidance creates access gap in UK for GPP drug Spevigo

FDA green lights CSL's once-monthly HAE drug Andembry

Sanofi draws up $9.1bn plan to buy Blueprint Meds

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