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- PMLiVE

Health Canada

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Ipsen to acquire rare disease specialist Albireo in deal worth over $950m

The deal will strengthen the biopharma’s liver disease pipeline with the addition of Bylvay

- PMLiVE

Takeda therapy shows promise in rare blood clotting disorder

Phase 3 results show the enzyme replacement therapy reduced thrombocytopenia events by 60%

- PMLiVE

Ipsen’s cabozantinib receives NICE approval for advanced hepatocellular carcinoma

The treatment showed a significant improvement in overall survival versus placebo

- PMLiVE

Sarepta to assess imlifidase as pre-treatment to Duchenne muscular dystrophy gene therapy

The company submitted a Biologics License Application to the FDA for SRP-9001 in September

Cell and gene therapies as depicted via a DNA strand

The fast-paced future of cell and gene therapies

In this report, we interview patients, researchers, and biopharmaceutical leaders to explore the complex landscape for the development of cell and gene therapies and share commercial recommendations from our specialist...

Avalere Health

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FDA receives Santhera and ReveraGen’s application for Duchenne muscular dystrophy treatment

The submission includes positive data from the pivotal phase 2b and four open-label studies

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The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia

With transformative treatments that leverage CRISPR technology, there are many challenges to overcome throughout the journey of development through to patient access. We interviewed a researcher at a top 20...

Avalere Health

Overcoming Patient Recruitment Challenges in Rare Disease Trials

Rare diseases are thought to affect up to 446 million people worldwide. With more than 7,000 rare diseases, most of which are genetic, it’s vital all patients can access new...

Innovative Trials

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FREE Live Webinar – Overcoming Patient Recruitment Challenges in Rare Disease Trials

Innovative Trials and Pfizer invite you to attend the live webinar

Innovative Trials

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How whole genome sequencing is revolutionising the rare disease patient experience

In this white paper, we discuss how whole genome sequencing (WGS) projects and the data sets they produce are paving the way for developments in research, diagnosis, and early interventions...

IGNIFI

Democratizing Brain Health with Digital Assessments

Dr. Pam Ventola, Senior Science Director at Cogstate, shares how the company is working to optimize brain health assessments in rare disease and pediatric clinical trials, and how this can...

Impetus Digital

- PMLiVE

Improving healthcare for rare disease patients

Following on from Rare Disease Day 2022, we decided to take a deeper look into the patient journey of those living with a rare health condition. In this blog, we...

IGNIFI

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