Category: Uncategorised

HCU Easy Tablets

Effective 1st June 2020 HCU Easy Tablets will be listed on the Pharmaceutical Benefits Scheme (PBS). HCU Easy Tablets are a methionine free protein substitute tablet containing essential and non essential amino acids. See information sheet about HCU Easy Tablets here. Speak to your Metabolic Team if you have any enquiries regarding HCU Easy Tablets.

Interview with Dr. Ken Maclean

Dr. Maclean was the 1st Recipient of HCU Global Grant Research Award, an effort that is jointly sponsored by HCU Network America and HCU Network Australia to support research on potential advances in the diagnosis and treatment of classical HCU.  A grant of $40000 USD was provided to support his project to investigate the potential…

Aeglea BioTherapeutics Clinical Trial Approval

Aeglea BioTherapeutics has announced approval of their clinical trial application for its novel engineered human enzyme designed to treat homocystinuria. “The approval of the CTA for ACN00177 is an important step forward for our Homocystinuria program and for patients who are in need of new treatment options. Given these unprecedented times, our priorities are to…

National Strategic Action Plan for Rare Diseases

The National Strategic Action Plan for Rare Diseases (the Action Plan) was launched in 2020 after Australian academics first called for a national plan for rare diseases in 2010. Rare Voices Australia (RVA) has led the collaborative development of the Action Plan. After extensive consultation with stakeholders around the country, the Action Plan has been…

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 Australia today announced the second recipient of their CBS deficiency global grants program – awarding a research grant to the University of Colorado Anschutz Medical Campus in Aurora to explore a potential treatment for homocystinuria due to cystathionine beta-synthase (CBS) deficiency. The research,…

Remembering Prof. Jan Kraus

Professor Jan Kraus passed away on the morning of July 4th after a battle with cancer.  The research of Professor Kraus focused on propionic acidemia and classical homocystinuria.  He dedicated his professional life to the understanding, diagnosis and treatment of classical homocystinuria and proprionic acidemia. Professor Kraus was recognised internationally as a brilliant scientist and considered by many as the “Father…

Third International Patient Expert Meeting Rome 2019

What a fabulous meeting we had at the Third International Homocystinurias Patient Expert Meeting in Rome.  The program was packed with the latest research, clinical care and dietary management for these disorders.  We are most grateful to the researchers, clinicians and dieticians that attended and for their willingness to spend time and share their knowledge…

First HCU patients treated in Phase 1/2 of OT-58

Orphan Technologies, a company dedicated to helping patients control their homocysteine levels, has announced that the first patients with classical homocystinuria have been treated in a Phase 1/2 clinical trial of OT-58. OT-58 is a novel, recombinant enzyme therapy designed to reduce plasma and tissue homocysteine levels. We are fortunate to have both Dr. Marcia…

Clinical and Basic Investigations of MMA and related disorders

The National Institutes of Health (NIH) sponsored research study named Clinical and Basic Investigations of Methylmalonic Acidemia (MMA) will evaluate patients with MMA and related disorders to learn more about the genetic causes of the various types of these inherited metabolic disorders and the medical complications associated with them. Dr. Venditti and his research team…

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 of Medicine who has received a Research Grant from HCU Network Australia together with HCU Network America to explore new strategies for improving treatment of homocystinuria due to CBS deficiency and remethylation defects. Read more below. MEDIA RELEASE FOR RELEASE: 25 January 2019…