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…
Cystic fibrosis has proved resistant to treatments that provide long-lasting respite from the sticky mucus…
Lysosomal storage disorders such as Niemann-Pick type C have proved difficult to tackle in the…
New, more targeted treatments are on the horizon for the rare autoimmune disease ANCA-associated vasculitis,…
While individually uncommon, rare diseases collectively impact the lives of over 300 million people worldwide.…
The first RNA interference therapy was approved in 2018, after 20 years in development. What…
As the biotech industry flourishes and therapies become more targeted and personalized, the need for…
Update (19/05/2020): The gene therapy Zolgensma, developed by the big pharma Novartis and approved in…
The Italian biotech Newron is halting the development of the experimental drug sarizotan after it…
Update (02/04/2020): The FDA has concluded an investigation into a data manipulation scandal surrounding the…
The French pharmaceutical company Ipsen has restarted the dosing of its treatment for a rare…
More people are impacted by rare diseases than by cancer and AIDS combined. With 400…
The Dutch startup Azafaros has raised €25M in Series A funding to push its drug…