Bioblast Pharma

Hope should not be rare

Trial-related news archive

Trial-related News Archive

  • April 14, 2016
  • Bioblast Pharma
  • Trial-related news

Bioblast Pharma to Present Final HOPEMD Phase 2 Clinical Study Results of Trehalose in Oculopharyngeal Muscular Dystrophy at the American Academy of Neurology (AAN) 2016 Annual Meeting

Final Results of 24-Week Open-Label Phase 2 Trial Will be Presented by Professor Zohar Argov, MD, Senior Medical Advisor to Bioblast Pharma

NEW HAVEN, Conn., April 14, 2016 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, will present previously announced positive final results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD) at the 2016 American Academy of Neurology (AAN) Annual Meeting, being held in Vancouver, Canada April 15-21.  OPMD is a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk of aspiration of food into the lungs, weight loss, and generalized progressive muscle weakness.

American Academy of Neurology 2016 Annual Meeting
April 15-21 in Vancouver, Canada

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  • March 10, 2016
  • Bioblast Pharma
  • Trial-related news

BioBlast Pharma Announces Positive Final Results from HOPEMD Phase 2 Open-Label Clinical Study of Trehalose 90mg/mL IV Solution in Oculopharyngeal Muscular Dystrophy (OPMD)

NEW HAVEN, Conn., March 10, 2016 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today that Colin Foster, President and CEO, will provide a corporate overview and business update at the 28th Annual ROTH Conference, taking place in Orange County, California, March 13-16, 2016.

Presentation Details:

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  • October 27, 2015
  • Bioblast Pharma
  • Trial-related news

Bioblast Announces Positive Interim Results From HOPEMD Phase 2 Clinical Study of Cabaletta(R) in Oculopharyngeal Muscular Dystrophy (OPMD)

Conference Call and Webcast, 8:30 a.m. Eastern Time

NEW HAVEN, Conn. and TEL AVIV, Israel, Oct. 27, 2015 (GLOBE NEWSWIRE) — 

Investor
Presentation:

HOPEMD Interim Data

Bioblast Pharma Ltd., (Nasdaq:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced positive interim results from a Phase 2 open label clinical study of its lead compound, Cabaletta® (IV trehalose), in 25 patients with oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by severe swallowing difficulties (dysphagia) leading to malnutrition, dehydration, and aspiration of food into the lungs, as well as more generalized, progressive muscle weakness. Aspiration pneumonia and severe emaciation are frequently the cause of death.

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