Rare diseases

News

EMA finds possible link between mRNA COVID jabs and myocardi...

The EU medicines regulator has said that there is some evidence to suggest a possible link between mRNA-based vaccines for COVID-19 and rare cases of heart inflammation.

Views & Analysis

Doctor hand holding pen and talking to the patient about medication and treatment.

Rare disease: industry, patients and tech join forces on dia...

Takeda’s Adam Zaeske discusses how cross-sector collaboration is shaping a brighter future for rare disease diagnosis.

Views & Analysis

Where-next-for-patient-centricity-in-pharma

Why relationships are key in rare disease patient engagement

Real-world data is a vital part of rare disease drug development, but to get a true picture of patients’ unmet needs pharma should take a broader view of their lived experiences, says Xperi

Views & Analysis

Growing up different and not knowing why: My experience of r...

Protracted diagnostic odysseys don’t only block access to rare disease treatments, they also leave people facing emotional turmoil all alone.

Events

Partner Content

World Orphan Drug Congress USA Virtual 2021

World Orphan Drug Congress USA Virtual

News

Evrysdi has 'real world impact' on lives of babies with seve...

Roche’s Genentech unit has released further data from a key trial of Evrysdi, its oral treatment for spinal muscular atrophy (SMA), the rare muscle wasting disease that is often fatal in ba

EMA finds possible link between mRNA COVID jabs and myocardi...

Rare disease: industry, patients and tech join forces on dia...

Why relationships are key in rare disease patient engagement

Growing up different and not knowing why: My experience of r...

World Orphan Drug Congress USA Virtual 2021

Evrysdi has 'real world impact' on lives of babies with seve...

Halozyme abandons its €2bn pursuit of Evotec

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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