Pharmafile Logo
T40 2022 Cover
Diversity, Equity and Inclusion
Communiqué 53 Cover Image
- PMLiVE

Kyowa Kirin

- PMLiVE

Rare Disease Day – looking to the future with hope

On this year’s Rare Disease Day, Amanda Henkel, VP, Strategy, Nucleus X Consulting (part of Nucleus Global), reflects on recent advances in the rare disease setting.

Nucleus Global

- PMLiVE

SMC approves Parkinson’s disease treatment Ongentys

The once-daily additional oral treatment for patients living with Parkinson’s disease is now accepted for use within NHS Scotland

Developing Innovative Digital Strategies to Engage Patients

Caitlin Wagner, Associate Marketing Director at Pharming, shares her expertise on innovative patient engagement strategies, including the roles of mobile apps and social media, the impact of the COVID-19 pandemic...

Impetus Digital

Light Chain Amyloidosis

What does the future hold for Light-chain Amyloidosis?

Recent advances in the understanding and treatment are reforming pharma’s approach to the management of this rare disease. With a new standard of care rapidly developing, what does the landscape...

Research Partnership

- PMLiVE

bluebird bio gears up for split

The company has filled its coffers with $75m in new funding and hired new leaders to support its split into two separate companies, bluebird bio and 2seventy bio

- PMLiVE

CHMP rejects Kyowa Kirin’s Parkinson’s disease drug

Results of eight main studies of drug were 'inconsistent', according to the CHMP

- PMLiVE

Rare diseases: why they can be so difficult to identify

Symptoms shared with more common diagnoses can make it more difficult to spot underlying rare diseases

- PMLiVE

bluebird bio aiming to separate business units by year-end

Biotech company has revealed that its new, separate oncology company will be named 2seventy bio

- PMLiVE

Rare Disease Day in a COVID-19 environment

For many people and families living with a rare disease, their journey can be very complex. At times, that journey can even be scary, frustrating and confusing. However, when your...

Lucid Group Communications Limited

- PMLiVE

The five-step, rare disease-ready checklist

How can a commercial team find success in the rare disease area?

- PMLiVE

bluebird bio marks Rare Disease Day 2021 with launch of patient testimonial video

Personal stories from nine patients from seven European countries, aged between 4 and 61, shed light on the impact of living with thalassaemia

- PMLiVE

Protalix, Chiesi’s monthly treatment PRX-102 shows benefit in Fabry disease

Fabry disease is a rare disorder in which patients inherit a deficiency of the α–Galactosidase–A enzyme

Subscribe to our email news alerts

Latest jobs from #PharmaRole

Latest content

Latest intelligence

Quick links