BioSpace News Archive

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  Rare diseases

  Rare Disease Sales To Soar to $400B+ By 2032 as Small Molecules Resurgent: Evaluate

  Small-molecule drugs account for nearly half of the most valuable investigational therapies for orphan diseases, according to analytics firm Evaluate.

  March 13, 2026

   · 

  2 min read

   · 

  Tristan Manalac
• 

  Duchenne muscular dystrophy

  Solid Adds to Differentiated Profile With New Data for Duchenne Gene Therapy

  Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

  March 13, 2026

   · 

  2 min read

   · 

  Tristan Manalac
• 

  Editorial

  Prasad’s Request To Remain Anonymous Shines Light on FDA’s Transparency Problem

  While requests by government officials for anonymity when speaking to the media are nothing new, the practice attracts more scrutiny when the Department for Health and Human Services has pledged a commitment to “radical transparency.”

  March 13, 2026

   · 

  7 min read

   · 

  Heather McKenzie
• 

  Earnings

  Biotechs Report Regulatory Headaches, High-Stakes Catalysts During Q4 Earnings

  Dozens of biotechs reported earnings this week. BioSpace recaps key highlights from Capricor Therapeutics, Legend Biotech, Inovio and Allogene.

  March 13, 2026

   · 

  5 min read

   · 

  Annalee Armstrong
• Press Releases

  Celularity Reiterates Strategic Commercialization Partnership with NEXGEL Focused on Building Biomaterials Franchise with Established Commercial Products and New 510(k) Pathway Product Opportunities

  March 13, 2026

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  4 min read
• Press Releases

  Polaryx Therapeutics Deepens Engagement with the Krabbe Disease Community Through Scientific and Patient Advocacy Events

  March 13, 2026

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  6 min read
• Press Releases

  ORAGENICS INITIATES PHASE IIA CLINICAL TRIAL SITE ACTIVATION

  March 13, 2026

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  5 min read
• Press Releases

  Ultragenyx Announces Positive 36-Week Data from Phase 3 Study of DTX301 AAV8 Gene Therapy for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency

  March 13, 2026

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  9 min read
• Press Releases

  Baird Medical Expands Global Reach with Regulatory Clearance in Argentina

  March 13, 2026

   · 

  5 min read
• Press Releases

  Cresilon Announces Poster Presentation on TRAUMAGEL Case Study at the 2026 Advanced Trauma Life Support Global Symposium

  March 13, 2026

   · 

  2 min read