Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease Posted On: 11/08/2022 Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease Read More...
Small fibre involvement is independent from clinical pain in late-onset Pompe disease Posted On: 21/06/2022 A recent study has been carried out with the aim to analyse the pain characteristics and its relation to a small nerve fibre involvement in Late Onset Pompe patients. Read More...
What the development of Myozyme meant for patients, their families, and their community Posted On: 07/04/2022 The story of the UK Pompe community’s involvement in the development of enzyme replacement therapy for Pompe disease is the subject of an academic publication. Read More...
Invitation to UK patients to participate in research into patients' and families' experience of living with Pompe disease Posted On: 03/06/2021 An invitation for people in the UK living with Pompe disease to help with research which will explore the effects of Pompe disease on families. Read More...
First Patient Dosed in FORTIS Trial of AT845 for LOPD Posted On: 14/04/2021 The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). Read More...
Novel Oral Therapy for Pompe disease Posted On: 31/03/2021 A new player, MAZE Therapeutics, has joined the growing number of companies with an interest in Pompe disease. This time they are targeting Muscle Glycogen Synthase, the enzyme that causes a very rare muscle form of Glycogen Storage Type 0. Read More...
Spark Therapeutics Announces First Participant for LOPD Posted On: 01/02/2021 Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease. Read More...
AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipeline Posted On: 18/11/2020 - AVR-RD-03 for Pompe disease: Preclinical data show normalization of substrate levels in multiple hard-to-reach organs Read More...
MHRA deems Sanofi’s avalglucosidase alfa ‘promising’ for Pompe disease Posted On: 01/10/2020 The PharmaTimes online has reported that the MHRA have issued Sanofi a Promising Innovative Medicine designation in the treatment of Pompe disease. Read More...
UK Studies for Pompe disease Posted On: 08/05/2020 *Research Update - 17/09/20 There are a growing number of clinical studies for next-generation Enzyme Replacement Therapies, Gene Therapies. These are usually reported on US, EU and WHO websites, but we have selected those with UK study centres and will update our list to keep it relevant. Read More...
WORLD Symposium - Visit Report Posted On: 08/05/2020 Allan Muir recently attended the WORLD Symposium to hear of the latest research concerning Pompe disease and to meet privately with companies, researchers and patient groups to share experiences and hopes for the future. Read More...
Notes from SSIEM 2019, Rotterdam, 3-5 September 2019 Posted On: 08/05/2020 Allan Muir, Pompe UK, Allan Muir attended the 2019 symposium of the Society for Study of Inborn Errors of Metabolism (SSIEM) on behalf of the UK LSD Collaborative. Read More...
