News
Investigational Synthetic Biotic Medicine for the Treatment of Homocystinuria
Synlogic and Ginkgo have announced the nomination of SYNB1353, an investigational Sy…
Phase 1/2 COMPOSE Study of pegtibatinase
Travere Therapeutics have announced positive topline results from the ongoing Phase 1…
Australian Clinical Trial
Westmead Hospital is the first Australian site for a phase 1 clinical trial of an enz…
Homocystinuria Data Collection Program
Your participation in the Data Collection Program is one of the most important and cr…
Gene Therapy and Classical Homocystinuria
Our Scientific Advisory Board's co-chair, Professor Warren Kruger is pioneering gene …
Mind over Miles for Neve
The Whelan family are participating in the Race for Research this September, an initi…
Aeglea BioTherapeutics Doses First Patient
We are excited to see these clinical trials available for Australian patients. These …
Experiences of diagnosis and patient satisfaction
A paper from the HCU Network Australia survey on the experiences of the homocystinuri…
Aeglea BioTherapeutics Receives FDA Rare Pediatric Disease Designation for ACN00177 for the Treatment of Homocystinuria
Aeglea BioTherapeutics, Inc.has announced the U.S. Food and Drug Administration (FDA)…
HCU Easy Tablets
Effective 1st June 2020 HCU Easy Tablets will be listed on the Pharmaceutical Benef…
Interview with Dr. Ken Maclean
Dr. Maclean was the 1st Recipient of HCU Global Grant Research Award, an effort that …
Aeglea BioTherapeutics Clinical Trial Approval
Aeglea BioTherapeutics has announced approval of their clinical trial application for…
National Strategic Action Plan for Rare Diseases
The National Strategic Action Plan for Rare Diseases (the Action Plan) was launched i…
MEDIA RELEASE: Second recipient of the CBS deficiency global grants program released
MEDIA RELEASE FOR RELEASE: 6 March 2020 HCU Network America and HCU Network Aust…
Remembering Prof. Jan Kraus
Professor Jan Kraus passed away on the morning of July 4th after a battle with cancer…
Third International Patient Expert Meeting Rome 2019
What a fabulous meeting we had at the Third International Homocystinurias Patient Exp…
First HCU patients treated in Phase 1/2 of OT-58
Orphan Technologies, a company dedicated to helping patients control their homocystei…
Clinical and Basic Investigations of MMA and related disorders
The National Institutes of Health (NIH) sponsored research study named Clinical and B…
MEDIA RELEASE: First recipient of the CBS deficiency global grants program released
A very big congratulations to Professor Maclean of the University of Colorado School …
OT-58 as an Enzyme Replacement Therapy for Patients With Cystathionine Beta-Synthase Deficient Homocystinuria (CBSDH)
Phase 1 clinical trials of OT-58, an enzyme replacement therapy (ERT) in development …
Patient Information Booklet & Quick Guide
HCU Network Australia is excited to announce the release of two new resources…
The key to improving outcomes
An accurate diagnosis is the first step to improving the care and outcomes for those …
Experience of diagnosis and patient satisfaction
There is currently limited evidence showing the considerable length of time taken i…
Growth patterns in Irish B6 non responsive homocystinuria patients
At last year's International Congress of Inborn Errors of Metabolism (ICIEM) Orla Pu…
Early Detection is Key for Rare Diseases
Dr Tawfeg Ben-Omran, Senior Consultant, Paediatrics at Hamad Medical Corporations (HM…
Recap of 2017
On behalf of the team at HCU Network Australia, I would like to take the opportunity …
2018 CBS deficiency Research Grants
HCU Network Australia and HCU Network America are currently calling for Expressions o…
A innovative treatment approach for classical homocystinuria
At the recent International Congress of Inborn Errors of Metabolism (ICIEM) Erytech …
Developing an Enzyme Therapeutic for Homocystinuria
Matthew Bonem, et al. Poster presentation on developing an Enzyme Therapeutic for Ho…
2nd International Patient – Expert Meeting 2017, Rio de Janeiro
HCU Network Australia was delighted to collaborate again with the European Network an…
Seeking a cure for her son’s rare disorder
A Montana State University doctoral student seeking a cure for her son’s rare genet…
Testing erymethionase in CBS-deficient mice
Erytech Pharma, a French clinical-stage biopharmaceutical company developing innovati…
Hike4HCU
On 25th February, Team HCU embarked on an unforgettable 4 day and 3 night trek along …
ACNC Tick of Charity Registration
HCU Network Australia is proud to display the ACNC Tick of Charity Registration. Whe…
International Rare Disease Day 2017
The 28 February 2017 marks the tenth international Rare Disease Day coordinated by …
Natural History Study of CBS Deficiency
Orphan Technologies Ltd is looking for male and female patients, between the ages 5 t…
Clinical Guidelines for CBS Deficiency
The European Guidelines for the diagnosis and management of cystathionine beta-syntha…
Orphan Europe’s New Infographic
Oprhan Europe’s HCU infographic video for Healthcare Professionals provides an over…
CBS enzyme replacement therapy
Recent study suggests that CBS enzyme replacement therapy (ERT) is a promising approa…
Thank you Burnside Lodge and Masonicare
We were delighted for the opportunity to present to the Burnside Lodge members this w…
World Rare Disease Day 2016: A community for progress
HCU Network Australia was proud to jointly convene the international patient and expe…
Take a survey!
Homocystinuria Patient & Caregiver Survey: Experiences of Diagnosis and Beyond W…
Brazilian Homocystinuria Meeting of Patients and Families
We are pleased to share on behalf of Professor Ida Schwartz and the organisers the fi…
Welcome to scientific advisors
We are delighted to welcome our scientific and medical advisors: Prof Bridget Wilcken…
RRD Foundation Homocystinurias Course
The Recordati Rare Diseases Foundation will offer a Homocystinurias course in 2016. T…
Thank you Orphan Technologies
We are able to accelerate our impact and ensure we achieve our mission because of ong…
Thank you Team HCU
On September 20 Team HCU took part in the Sydney Running Festival to raise awareness …
HCU Patient Diagnosis Bags
We are delighted that our HCU patient diagnosis bags have now been distributed to the…
Cystadane on PBS effective 1 August 2014
Cystadane has been added to the Schedule of Pharmaceutical Benefits effective 1 Augus…
Clinical trials to commence for ERT for Classical Homocystinuria
The University of Colorado (CU) has signed exclusive, worldwide licensing and collabo…
Study discovers how rare disease causing cardiovascular and cognitive disorders is triggered
A study has discovered the molecular mechanism that triggers homocystinuria, which is…
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