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rare disease

  • Clinical & Regulatory,Drug Delivery & Formulation,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    FDA grants Priority Review to Belite Bio’s tinlarebant for Stargardt disease

    The FDA has accepted Belite Bio’s application for tinlarebant and [...]

    August 13, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Vaderis raises $152M and starts Phase 3 trial of engasertib in HHT

    Vaderis Therapeutics has raised $152M and started a global Phase [...]

    August 12, 2026
  • Central Nervous System,Clinical & Regulatory,Research & Development,Therapeutic Areas

    Zilucoplan accepted for eligible myasthenia gravis patients in NHS Scotland

    Zilucoplan has been accepted for restricted use in NHS Scotland [...]

    August 11, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    Grifols completes Phase 3 SPARTA study of Prolastin-C in alpha-1 emphysema

    Grifols has completed patient follow-up in its Phase 3 SPARTA [...]

    August 11, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development

    Amorphical completes Phase 2 enrolment for AMOR-1 in hypoparathyroidism

    Amorphical has completed patient enrolment in a Phase 2 trial [...]

    July 29, 2026
  • Clinical & Regulatory,Drug Delivery & Formulation,Pharmaceuticals and therapeutics,Research & Development

    FDA allows Medicus Pharma to start SkinJect study in patients with Gorlin Syndrome

    Medicus Pharma has received FDA clearance to begin a Phase [...]

    July 28, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    CAMP4 wins Australian clearance to begin Phase 1/2 trial of SYNGAP1 therapy

    CAMP4 Therapeutics has received Australian regulatory and ethics clearance to [...]

    July 27, 2026
  • Central Nervous System,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    CHMP recommends approval of NEZGLYAL for rare neurodegenerative disease

    The EMA’s CHMP has recommended approval of NEZGLYAL for boys [...]

    July 24, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Skyhawk expands global Huntington’s disease trial following positive Phase 1/2 data

    Skyhawk Therapeutics has expanded its global pivotal Phase 2/3 clinical [...]

    July 14, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Grifols doses first patient in Phase 3 alpha-1 therapy trial

    Grifols has dosed the first participant in a Phase 3 [...]

    July 2, 2026
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