Rare diseases

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Partner Content

Real-world evidence: breaking boundaries in rare disease

The COVID-19 pandemic has added a new layer of complexity to the drug development process.

News

bluebird bio to split into oncology and gene therapy special...

US biotech bluebird bio has announced plans to split into two this year, with a separate oncology business spinning off as the company prepares to bring its products to market.

Views & Analysis

2020 review - Pharma’s progress outside of COVID-19

2020 in review: Pharma’s progress outside of COVID-19

It has been a year dominated by the pandemic and many life sciences research projects were put on hold as big pharma turned its attention to vaccines and therapies.

R&D

Tay-Sachs and rare disease registries – the Health Heroes po...

Episode four of Kantar Health’s Health Heroes podcast tackles pharma’s rare disease challenges, the role of patient registries and hears a moving story about the rare genetic disorder Tay-S

Events

Partner Content

Operationalize: Expanded Access Programs Summit

Operational Expanded Access Programs Summit

News

Big Heavy Rocket Space Launch System Launch. 3D Illustration.

Rocket Pharmaceuticals in orbit after gene therapy read-out

Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare hea

Real-world evidence: breaking boundaries in rare disease

bluebird bio to split into oncology and gene therapy special...

2020 in review: Pharma’s progress outside of COVID-19

Tay-Sachs and rare disease registries – the Health Heroes po...

Operationalize: Expanded Access Programs Summit

Rocket Pharmaceuticals in orbit after gene therapy read-out

Ambrosia's sweet $100m round, and other biofinancings

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

Rare diseases

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