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Rare disease

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News and Trends

Biotechs Carve out Niches in Cystic Fibrosis Treatment Space

Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus…

By Anita Chakraverty
22/03/2022 - 5 minutes
News and Trends

Emerging Startups Take Aim at Lysosomal Storage Disorders

Lysosomal storage disorders such as Niemann-Pick type C have proved difficult to tackle in the…

By Jonathan Smith
10/03/2022 - 4 minutes
News and Trends

Targeted Drugs for Rare Autoimmune Disease Hit Regulatory Milestones

New, more targeted treatments are on the horizon for the rare autoimmune disease ANCA-associated vasculitis,…

By Anita Chakraverty
01/12/2021 - 4 minutes
Sponsored by Merck

Gene therapy: Making Rare Disease Therapies Less Rare

While individually uncommon, rare diseases collectively impact the lives of over 300 million people worldwide.…

By External Contributor
15/11/2021 - 2 minutes
In Depth

The Coming of Age Story of RNAi Technology

The first RNA interference therapy was approved in 2018, after 20 years in development. What…

By Farhan Mitha
02/06/2021 - 6 minutes
Expert Advice

Why Listening to Patient Organizations is Key in Drug Development

As the biotech industry flourishes and therapies become more targeted and personalized, the need for…

By Larissa Warneck-Silvestrin
27/05/2020 - 11 minutes
News and Trends

Update: Novartis’ Gene Therapy Zolgensma Gets Nod from EU

Update (19/05/2020): The gene therapy Zolgensma, developed by the big pharma Novartis and approved in…

By Jonathan Smith
19/05/2020 - 5 minutes
News and Trends

Phase III Failure Sinks Drug for Rare Neurological Disorder

The Italian biotech Newron is halting the development of the experimental drug sarizotan after it…

By Anita Chakraverty
08/05/2020 - 4 minutes
News and Trends

Update: FDA Imposes No Penalties for Novartis Data Manipulation Scandal

Update (02/04/2020): The FDA has concluded an investigation into a data manipulation scandal surrounding the…

By Jonathan Smith
02/04/2020 - 3 minutes
News and Trends

Ipsen Resumes Phase III Testing for Rare Disease Drug and Abandons Pediatric Trial

The French pharmaceutical company Ipsen has restarted the dosing of its treatment for a rare…

By Karen O’Hanlon Cohrt
31/03/2020 - 4 minutes
Sponsored by CENTOGENE

How Can We Improve the Diagnosis and Treatment of Rare Diseases?

More people are impacted by rare diseases than by cancer and AIDS combined. With 400…

By Larissa Warneck-Silvestrin
17/02/2020 - 7 minutes
News and Trends

First-in-Class Drug For Inherited Metabolic Diseases Funded with €25M

The Dutch startup Azafaros has raised €25M in Series A funding to push its drug…

By Jonathan Smith
10/02/2020 - 2 minutes
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