Shining a light on those living with sickle cell disease
Charity groups and campaigners are raising awareness of sickle cell disease as those living with the most common genetic condition in the UK continue to face barriers to healthcare access. Sickle cell disease (SCD) is the most common genetic condition in the UK but people living with SCD still face barriers accessing care and treatment. … Continued
Rare Diseases Q1 2022
Adoption of UN resolution is huge triumph for rare disease community
A ground-breaking resolution from the United Nations finally gives visibility to 300 million persons living with a rare disease (PLWRD) and their loved ones worldwide. To mark Rare Disease Day, the global community celebrates the adoption of the first-ever UN resolution on “Addressing the Challenges of Persons Living with a Rare Disease and their Families.” … Continued
How novel companies are leading the way in orphan drug strategies
What makes a company successful in orphan drug development? When orphan drugs first came to notice in the late 1990s, pharma companies saw insufficient commercial potential. Small inexperienced companies, on occasion born out of desperation of families afflicted by a rare disease, have led the way in orphan drug development. These small companies therefore require … Continued