The coming of age story of RNAi technology
The first RNA interference (RNAi) therapy was approved in 2018, after 20 years in development.…
July 22, 2022 - 7 minutes
Rare disease
Almirall launches new call to find innovative therapies for skin diseases
A new call for new therapies to treat skin diseases has been launched by global…
July 21, 2022 - 3 minutes
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…
March 22, 2022 - 5 minutes
Emerging Startups Take Aim at Lysosomal Storage Disorders
Lysosomal storage disorders such as Niemann-Pick type C have proved difficult to tackle in the…
March 10, 2022 - 4 minutes
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,…
December 1, 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.…
November 15, 2021 - 2 minutes
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…
May 27, 2020 - 11 minutes
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…
May 19, 2020 - 5 minutes
Phase III Failure Sinks Drug for Rare Neurological Disorder
The Italian biotech Newron is halting the development of the experimental drug sarizotan after it…
May 8, 2020 - 4 minutes
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…
April 2, 2020 - 3 minutes
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…
March 31, 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…
February 17, 2020 - 7 minutes
