Weekly Spotlight - 31.10.24

In the News

Tyra Biosciences Advances TYRA-300 for Achondroplasia Treatment in Children

Tyra Biosciences has received FDA clearance to begin a Phase 2 study of TYRA-300 for children with achondroplasia. This oral treatment aims to improve growth and quality of life. The study will assess safety and effectiveness, offering hope for better therapeutic options for this rare condition.

BioMarin's Q3 Success Despite Voxzogo's Slight Shortfall

BioMarin exceeded Q3 sales expectations with £746 million in revenue, a 28% increase, despite Voxzogo missing its target by 3%. The achondroplasia treatment saw a 54% sales rise. Roctavian, a new gene therapy, contributed £7 million, showing promising growth potential in key markets like the US, Italy, and Germany.

Dwarfism Awareness: Celebrating Diversity and Understanding Achondroplasia

International Dwarfism Awareness Day, celebrated on 25th October, highlights the condition of dwarfism, particularly achondroplasia, a common bone growth disorder. Founded by Little People of America in 2012, the day promotes understanding and acceptance, emphasising that individuals with dwarfism lead fulfilling lives and are capable of achieving their goals.

Quaden Bayles: A Beacon of Resilience and Community Support

Quaden Bayles, a young boy with achondroplasia, has become a global symbol of resilience and anti-bullying advocacy. His viral story highlights the importance of kindness and community support. Quaden's advocacy work and public speaking have inspired many to promote inclusivity and empathy, fostering a more compassionate society.

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