Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Pfizer has initiated a Phase 3 trial for giroctocogene fitelparvovec (PF-07055480), a gene therapy targeting severe hemophilia A. This advancement reflects Pfizer's commitment to developing transformative treatments for rare diseases.
Read full storyCongress has passed a landmark bill aimed at protecting patient access to specialty pharmacies, crucial for managing complex health conditions such as cancer and rare diseases. This legislation is expected to stabilize the specialty pharmacy infrastructure, enhancing innovation and patient outcomes.
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