Rare Disease Day at NIH highlights the collaboration between the Rare Diseases Clinical Research Network and patient advocacy groups. This event aims to raise awareness and support for rare diseases through community engagement.

Rare Disease Day emphasizes the critical role of patient advocacy groups in driving research and treatment development for rare diseases. Celebrated annually on the last day of February, it aims to raise awareness and mobilize resources for conditions that often receive less attention.

Angela, Founder and President of Cal Rare, has significantly influenced rare disease advocacy in California, leading to the formation of the California Rare Disease Legislative Caucus, which now includes 25% of the State Legislature. Her recognition includes the Rare Voice Award for State Advocacy and accolades for her social media impact on rare disease awareness.

Amgen acquires Horizon Therapeutics for $27.3 billion, highlighting significant consolidation in the rare disease sector amid challenging financial conditions. This acquisition underscores the ongoing interest in rare disease therapies despite market difficulties.

Chiesi Farmaceutici acquired Amryt Pharma for up to $1.5 billion, while Ipsen purchased Albireo for up to $1.2 billion, marking a strong start for M&A in the rare disease sector in 2023. Despite these significant deals, overall financing in the sector remains weak.

The recent government funding bill reauthorizes the rare pediatric disease voucher program, crucial for accelerating drug development for rare childhood diseases. This bipartisan effort, highlighted by the Mikaela Naylon Give Kids a Chance Act, aims to support families affected by these conditions.

FDA has placed a clinical hold on Regenxbio's gene therapy RGX-111 due to the discovery of a brain tumor in a trial participant. This decision has led to a 30% drop in the company's shares, highlighting significant concerns regarding the safety of the treatment for rare childhood diseases.