Nathalie Goemans, MD, PhD | DuchenneXchange

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Researchers

Nathalie  Goemans, MD, PhD

Researcher
Head of the Neuromuscular Reference Centre
Department of Paediatrics and Child Neurology
University Hospitals Leuven
Campus Gasthuisberg
Herestraat 49
Leuven, Belgium

Dr. Nathalie Goemans is the head of the Neuromuscular Reference Centre within the department of Paediatrics and Child Neurology at the University Hospitals Leuven, Belgium. She is a consultant in neuropediatrics at DVC St Jozef Antwerp, Belgium, a rehabilitation centre with residential setting for neuromuscular patients.

Dr. Goemans is a pediatric neurologist with certification in rehabilitation medicine. She has special interests in the diagnosis and multidisciplinary aspects of care and management of paediatric neuromuscular disorders in general. She has developed a particular interest in the dissemination of standard of care and ethical issues in NMD, as well as in the design and conduct of clinical trials in neuromuscular disorders, more particularly in DMD and SMA.

Dr. Goemans has participated in development and dissemination of international guidelines for diagnosis and treatment of NMD and has longstanding experience in clinical research in NMD. Nathalie is actively involved in the development of protocols for international multicentre trials and is investigator of ongoing clinical trials in DMD such as PRO051-02: phase 1/2, PRO 044 phase ½, GSK DMD 114044, PTC124-GD-007-DMD: phase 2b, and the TROPHOS study in SMA.

 

Representative Publications:

Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM

Ataluren treatment of patients with nonsense mutation dystrophinopathy

Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy

Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

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