Rare diseases

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Alexion joins with Dicerna to develop RNAi therapies for com...

Alexion has announced a collaboration with Dicerna Pharmaceuticals to discover and develop RNA interference therapies for diseases caused by problems in the complement system.

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

UK biotech Orchard plans $172.5m US IPO to fund rare disease...

UK biotech Orchard Therapeutics is planning a US IPO to expand use of its Strimvelis gene therapy, and further develop its rare disease pipeline.

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Preliminary Attendee List Released for Orphan Drugs & Rare D...

Attendees Released for Orphan Drugs & Rare Diseases

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FDA approves Ionis/Akcea's Tegsedi RNAi drug, taking on Alny...

The FDA has approved Ionis Pharmaceuticals’ RNA silencing drug Tegsedi (inotersen) for nerve damage caused by a rare disease.

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Roche's Hemlibra approved in expanded haemophilia A use

Roche’s Hemlibra has been approved in an expanded use in haemophilia A in the US, as the Swiss firm seeks to build a new blockbuster franchise in rare diseases.

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Denmark funds Vertex's CF drug Orkambi, UK talks deadlocked

As uncertainty hangs over the availability of Vertex’s cystic fibrosis drug in England, the company has signed an agreement that gives Danish patients access to Orkambi.

Alexion joins with Dicerna to develop RNAi therapies for com...

UK biotech Orchard plans $172.5m US IPO to fund rare disease...

Preliminary Attendee List Released for Orphan Drugs & Rare D...

FDA approves Ionis/Akcea's Tegsedi RNAi drug, taking on Alny...

Roche's Hemlibra approved in expanded haemophilia A use

Denmark funds Vertex's CF drug Orkambi, UK talks deadlocked

Biotech is back, with Craig Ackerman

Using AI-driven synthetic personas to take your insights fur...

Rare diseases

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