- There is considerable interest, excitement and anticipation for the development of adeno associated virus (AAV) gene therapy as a treatment for Duchenne muscular dystrophy (DMD). Much of this expectation is based on promising preclinical data as well as success in other neuromuscular conditions, such as spinal muscular atrophy (SMA). However, it is also important to take an objective view of the realistic possibilities and limitations of AAV gene therapy for DMD as well as considering the likely barriers to clinical trials and the development of gene therapy as an approved and accessible treatment.
- The 249th ENMC workshop was held from 29th November to 1st December 2019, and brought together 24 representative of patients, neuromuscular, neuropsychology and psychiatry experts from Europe, the United States and Japan. The topic of this workshop was the brain co-morbidities in Duchenne and Becker muscular dystrophies (DMD and BMD respectively).
- The European Neuromuscular Centre (ENMC) has always supported the use of ventilatory support in neuromuscular disorders (NMD's). There is a lack of provision of mouthpiece ventilation (MPV) and a lack of consensus with regard to how/when it can be delivered, The ENMC convened the 252nd ENMC International Workshop: Developing best practice guidelines for management of mouthpiece ventilation in Neuromuscular Disorders in Amsterdam, The Netherlands from the 6th to 8th March 2020. A total of 22 participants, 20 from Europe, one form the USA and one from Canada representing a total of 11 countries.
- Thirty-six representatives of academia, clinics, industry and patient organisations from 10 countries (Belgium, the Netherlands, Spain, Italy, Greece, France, United Kingdom, Switzerland, United States, Canada) attended the ‘Meeting on data sharing for Duchenne’ organised by the Duchenne Parent Project. During the meeting the current status, opportunities and challenges of data sharing in the Duchenne field were discussed.
- Twenty-six representatives of academia, clinics, patient organisations and industry from eight countries (Argentina; Australia; Belgium; Italy; the Netherlands; Switzerland; United Kingdom; United States) attended the workshop on ‘Nutrition in Duchenne muscular dystrophy’ organised by the Duchenne Parent Project the Netherlands (DPP). During the workshop current knowledge and clinical practice regarding nutrition in DMD was discussed, as well as research, preclinical and clinical, and guidelines for patients and caretakers.
- Thirty-one participants from 7 countries (Australia; England; France; Germany; Italy; The Netherlands; USA) attended the second ENMC workshop on antisense oligonucleotides in Duchenne muscular Dystrophy (DMD). The topic of this workshop was on “Planning Phase I/II Clinical Trials Using systemically delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy (DMD)” and followed a similar workshop held in 2004 focused on intramuscular administration of antisense oligonucleotides or AONs. The workshop was organized with the support of the TREAT-NMD EU Network of Excellence ( www.treat-nmd.eu ) and Parent Project Muscular Dystrophy (PPMD), and was attended by representative of the two companies involved in the current intramuscular injection trials, Prosensa for the 2-O-methyl phosphorothioate RNA modified and AVI Biopharma for the phosphorodiamidate morpholino oligomers (PMOs or more commonly “morpholinos”).
- Eighteen participants representing clinicians, scientists, parents, industry, and public health agencies involved in the assessment and treatment of children with Duchenne muscular dystrophy (DMD) from Australia, Canada, the Netherlands, the UK and the USA met in Philadelphia on December 8–9, 2006 to attend a workshop addressing behavioral issues among children and young adults with DMD. The workshop, sponsored by Parent Project Muscular Dystrophy (PPMD–USA), was organized in response to parent and clinician concerns regarding behavior associated with DMD, and its subsequent impact on patient and family quality of life.