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rare disease

This page shows the latest rare disease news and features for those working in and with pharma, biotech and healthcare.

FDA priority review for Roche’s Esbriet in unclassifiable interstitial lung disease

FDA priority review for Roche’s Esbriet in unclassifiable interstitial lung disease

Interstitial lung disease (ILD) encompasses a diverse group of over 200 types of rare pulmonary diseases. ... We are working closely with the FDA in the hopes of offering Esbriet to people with UILD, a rare and debilitating disease,” he added.

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  • Real-world evidence: breaking boundaries in rare disease

    However, small patient populations, the high unmet need, and difficulties identifying a control group make it difficult to conduct an RCT when trialling a rare disease therapy. ... Sonal Bhatia, Vice President, Medical Affairs Rare Disease at Pfizer,  

  • Unpacking rare diseases in the first edition of Delta magazine

    Fishawack Health launches Delta magazine with a deep dive into rare disease commercialization. ... Navigating real-world evidence and why it could be a gamechanger in rare disease.

  • emotive supports Rare Disease Day 2019

    With a series of events and activities over the week leading up to Rare Disease Day on February 28th, we showed our support for stimulating greater awareness of Rare Diseases along ... In line with the Rare Disease Day theme, we took photos of our team

  • Porphyria Awareness Week – Round Up

    Porphyria Awareness Week has given the team at emotive an opportunity to show our support for those touched by this debilitating Rare Disease as well as our enthusiasm for working in ... Porphyria is a rare condition that is thought to affect around 1 in

  • Learning about Rare Genetic Disorders on Undiagnosed Children’s Day

    Children can be left facing an uncertain future and an unclear treatment pathway when they have a rare disorder. ... A   Rare Disease UK  report published in 2016 found that nearly half of the respondents who identified as undiagnosed had been waiting

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Mistrust in medical research: a patient perspective
The recent development of several COVID-19 vaccines has placed medical research firmly in the spotlight, highlighting public confusion and misinformation about clinical trials. Patient advocate, Trishna Bharadia reveals what the...
Real-world evidence: breaking boundaries in rare disease
Generating data for drug launches is a challenging process. In rare diseases, with small patient populations and high unmet need, evidence generation is even more complex. Consultant Sarah Poole and...
The heavy toll of COVID-19 on cancer patients
We all know that finding and treating cancer early saves lives. During the COVID-19 pandemic, oncology treatments paused while cancer continued to spread. So, what has been the impact of...

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