BioMarin announced an additional serious adverse event in its gene therapy clinical trial for haemophilia A

September 13, 2022 – BioMarin reported today an update on the phase 3 clinical trial, which contained information on an additional serious adverse event (SAE) in a participant from the clinical trial with Roctavian™ (valoctocogene roxaparvovec). A serious adverse event (SAE) is the term used to describe the occurrence of a serious health issue in [...]

Meet Jeanette Fava, EHC Operations Director

We are delighted to introduce you to Jeannette Fava, EHC's Operations Director. Jeanette joined the EHC in June 2022 and will be overseeing the EHC's financial and administrative activities as well as managing the EHC's human resources. Here below we give you a quick interview of Jeanette so that you can learn more about her [...]

Update on the Recall of Desmopressin Nasal Sprays

The European Haemophilia Consortium (EHC) would like to update its February 2021 statement concerning the international recall of all Ferring Pharmaceutical Limited nasal sprays containing desmopressin, including Octim®, Octostim®, Desmospray®, DDAVP Spray® and Stimate®. On 31 May 2022, Ferring Pharmaceuticals Limited informed the EHC that the above products would not be back onto the European […]

BioMarin follows up on previously-reported  serious adverse event in its phase I/II gene therapy clinical trial for haemophilia A

May 5, 2022 – This is an update to the EHC’s February 4, 2022 statement regarding ‘BioMarin reporting a serious adverse event for its phase I/II gene therapy trial with valoctocogene roxaparvovec for haemophilia. Valoctocogene roxaparvovec is an experimental gene therapy (adeno-associated virus [AAV] delivering the B-domain deleted factor VIII gene) under development by BioMarin [...]

Pfizer/Sangamo Therapeutics report severe adverse event (SAE) from phase 3 AFFINE haemophilia A gene therapy study

May 5, 2022 – Pfizer/Sangamo have reported a severe adverse event (SAE) learning of a below-the-knee deep vein thrombosis (DVT) in one participant of its giroctocogene fitelparvovec clinical trial, who had elevated Factor VIII levels. A serious adverse event (SAE) is the term used to describe the occurrence of a serious health issue in a [...]

Meet Zita Gacser, EHC Think Tank Officer

We are delighted to introduce you to Zita Gacser, EHC Think Tank Officer. Zita joins Naja Skouw-Rasmussen in the Think Tank team and will be responsible for communications activities related to the Think Tank, supporting the development of the EHCucate app and supporting hybrid events.  Zita, can you tell us about yourself? I originally come [...]

Announcing: EHC Emergency Displacement Fund (EDF)

With enduring shock and sadness, we think of our community members and others affected by the current challenging times, and are working tirelessly to provide support where possible. The EHC have been working with our NMOs in the neighbouring countries and region to identify their needs, understand the key areas of support we may provide, […]

EHC Women’s Committee marks International Women’s Day

To mark the 2022 International Women's Day, held on 8 March, the EHC Women and Bleeding Disorders Committee wishes to share a pleasant surprise! The Committee has prepared an 'empty' copy of its 'diagnosis-quiz flyer (see below) so that EHC members can fill it in their local language and distribute it amongst their membership. This flyer can [...]

EHC VWD Working Group holds a strategic retreat

It was amongst much excitement and trepidation that the EHC von Willebrand Disease (VWD) Working Group (WG) finally met face-to-face in Dublin, Ireland, on Friday 28 January 2022. The EHC VWD Working Group was established in February 2020 to lead the work of the EHC VWD Platform for Europe. Still, due to the circumstances we [...]

Meet Daria Mironova, EHC Communications and Public Policy Officer

We are delighted to introduce you to our latest EHC addition, Daria Mironova. Daria joined us in December as Communications and Public Policy Officer. For this edition of our NMO Newsletter, we wish to tell you more about Daria and her role within the EHC.  Daria, can you tell us about yourself?  Yes, of course. [...]