News

Those with Achondroplasia are at a Higher Risk of Hight Blood Pressure and Heart Disease

We’re halfway through February! Which means, we’re halfway through Rare Disease Month! In honor of Valentine’s Day, this post focuses on heart health. Did You Know that February is also American Heart Month? Due to…

Rare Disease Day

Any Disease Affecting Fewer Than 200,000 People in the U.S. is Considered Rare

Any disease affecting fewer than 200,00 people in the United States is considered rare. Today, 25+ million Americans are living with a rare disease. Did You Know that 1 in 25,000 children are born with achondroplasia?…

Rare Disease Day

70% of Genetic Rare Diseases Start in Childhood

70% of genetic rare diseases start in childhood. Many rare diseases may result in the premature death of infants or can be fatal in early childhood. 80% of children with…

Rare Disease Day

Kickoff to Rare Disease Month

Did you know that rare diseases affect an estimated 350 million people worldwide? According to the National Organization for Rare Disorders, achondroplasia, the most common form of dwarfism, is considered…

Pfizer · Therachon

Pfizer Cuts Recifercept for Achondroplasia Program

Pfizer punts pair of orphan drugs amid rare disease R&D rethink Pfizer, amid a rethink of its rare disease R&D strategy, has punted (PDF) a pair of orphan drug candidates from its…

BioMarin · Voxzogo

European Medicines Agency Validates Application for Extension of Indication for VOXZOGO® (vosoritide) for injection to Treat Children with Achondroplasia Under the Age of 2

BioMarin Submits Supplemental New Drug Application to U.S. Food and Drug Administration to Expand Label to Treat Children with Achondroplasia Under the Age of 5 Submissions Based on Favorable Results…