Rocket Pharmaceuticals Advances RP-A501 Toward Danon Disease Approval
CRANBURY, N.J. — September 15, 2026 Rocket Pharmaceuticals, Inc. announced alignment with the U.S. Food and Drug Administration (FDA)...
rare disease
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Lundbeck Completes Enrollment in Phase III Dravet Trial
Copenhagen, Denmark, September 15, 2026 H. Lundbeck A/S has announced that the last patient has been randomized in DEEp...
Tenaya Therapeutics Gains RMAT Designation for TN-401
SOUTH SAN FRANCISCO, Calif. — September 14, 2026 Tenaya Therapeutics, Inc. announced that the U.S. Food and Drug Administration...
Sionna Advances SION-451 and SION-2222 Into Phase 2a
WALTHAM, Mass. — September 14, 2026 Sionna Therapeutics, Inc. announced plans to advance its preferred proprietary dual combination of...
Quantum BioPharma Advances Lucid-MS Toward Phase 2 in MS
NEW YORK — September 11, 2026 Quantum BioPharma Ltd. announced that the U.S. Food and Drug Administration has cleared...
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Pharming Gains FDA Approval for Pediatric Joenja in APDS
LEIDEN, Netherlands — September 11, 2026 Pharming Group N.V. announced that the U.S. Food and Drug Administration (FDA) has...
Lundbeck Gains FDA Orphan Drug Status for Asedebart
Copenhagen, Denmark, September 10, 2026 H. Lundbeck A/S has announced that the U.S. Food and Drug Administration (FDA) has...
Kazia Doses First Patient in Pediatric AT/RT Study
Sydney, Australia, September 10, 2026 Kazia Therapeutics Limited has announced that the first patient has been dosed in the...
Rezolute Awaits FDA Review of Ersodetug in Congenital HI
REDWOOD CITY, Calif. — September 9, 2026 Rezolute, Inc. provided an update on the U.S. Food and Drug Administration’s...
BioMarin Reports Positive Phase 3 VOXZOGO Data
San Rafael, California, September 9, 2026 BioMarin Pharmaceutical Inc. has announced the publication of positive Phase 3 CANOPY-HCH-3 results...
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