Updates

Orphazyme Appoints Christophe Bourdon as Chief Executive Officer

Copenhagen, Denmark, March 1, 2021 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announced the appointment of Christophe Bourdon as the company’s Chief Executive...

Cyclo Therapeutics Unveils New Corporate Identity and Reaffirms Commitment to Improving Quality of Life and Providing Hope for Patients and Families

17 de febrero Día Nacional de Niemann-Pick – Fundación Niemann-Pick de España

Julia González, Coordinadora familiar, at Fundación Niemann-Pick de España provided the INPDA with the charity’s official statement for National Niemann-Pick Day, which takes place today on February 17th 2021. “…one of the ways we have to raise awareness in...

Cyclo Therapeutics Presents Positive Data from Clinical Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

Data from ongoing Phase 1 extension study shows improvement in disease features or disease stabilization with home-based intravenous infusions of Trappsol® Cyclo™ Phase 1 biomarker 24S-hydroxycholesterol demonstrates clearance of excess cholesterol from the brain after intravenous infusions Interim analysis...

Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

Orphazyme US Investor news                                                                       ...

Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021

We are pleased to share the latest update from Cyclo Therapeutics entitled “Cyclo Therapeutics to Present on Its Clinical and Drug Development Program for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2021”:

A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia

We are pleased to share the following article “A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia” which describes the collaborative development of an innovative...

INPDA Spotlight on Niemann Pick Selbsthilfegruppe Deutschland e.V.

Sometimes in such a big organisation as the INPDA, our various member groups can be overshadowed, but we want to show just how important each and every member group is in making the INPDA work. In the INPDA Spotlight this...

Cyclo Therapeutics Announces Positive Efficacy Data from Extension Protocol with Trappsol® Cyclo™ in Patients with Niemann-Pick Disease Type C

Home-based intravenous infusions of Trappsol® Cyclo™ for up to one year show improvement in disease features or disease stabilization Gainesville, FL – (Businesswire) – 05 January 2021 – Cyclo Therapeutics, Inc. (NasdaqCM:CYTH and CYTHW), a clinical stage biotechnology company...

Orphazyme Provides Regulatory Update on Arimoclomol for NPC

Copenhagen, Denmark – December 27, 2020 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announced today the U.S. Food and Drug Administration (FDA) has extended the...