For its part, the Patients For Affordable Drugs Now (P4ADNOW), an organization focused on lowering drug prices, touted the exclusion of the Orphan Cures Act from the Senate’s reconciliation text ...

The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of sickle cell disease. Rilzabrutinib is an investigational, oral reversible Bruton ...

Beam Therapeutics rises after the FDA grants orphan drug status to its gene-editing therapy BEAM-101 for sickle cell disease.

EMA Orphan Drug Designation (ODD) builds on U.S. Food & Drug Administration (FDA) ODD and Fast Track Designation, underscoring the urgent need for new treatment optionsThe multiple regulatory ...

The FDA's orphan drug designation provides benefits like market exclusivity and tax credits, encouraging development for rare diseases affecting fewer than 200,000 people annually. Preclinical studies ...

IP University to introduce quota for orphan children from 2025-26, full fee waiver announced. Explore. Search Saturday, Jun 14, 2025. New Delhi o C. Subscribe. Games. E-Paper Sign in. My Account ...

Bail Reform Is an Orphan. Screen grab of former Governor Andrew Cuomo at the New York City mayoral debate, June 5, 2025. (NBC New York/YouTube) Share Comments Listen. By Noah Rothman.

Guru Gobind Singh Indraprastha University will introduce an "orphan quota" from 2025-26, reserving seats and offering fee waivers for affected students. | Latest News Delhi ...

Paul Verdin, VP of consulting and analytics, and Andreas Hadjivasiliou, managing analyst at Evaluate, tell us about the company’s Orphan Drug Report 2022, which highlights activities within the ...

The Well Done Foundation in Montana is collaborating with Millcreek Township and Pittsburgh students to plug an abandoned well in Erie County.

Circle Pharma Receives FDA Orphan Drug Designation for CID-078 for the Treatment of Small Cell Lung Cancer. Business Wire; Jun 16, 2025 Jun 16, 2025; 2 min to read.

Out of 51 applications received by the FDA Orphan Products Grants Program in fiscal year 2024, Palvella's Phase 3 Selva trial was one of only seven new clinical trials selected for funding—and ...