Growing the drug pipeline for neuromuscular diseases: optimising resources for
clinical development of new therapies


Welcome to Washington

We look forward to hosting you at the TREAT-NMD International Conference in Washington DC!


The Conference Committee has organized a very current and translational agenda - orphan drug development in neuromuscular disease is 'heating up' and there is a lot to catch up on.  As previously, the conference is designed with ample time for informal interactions with speakers and your colleagues.


This is the first TREAT-NMD Conference to be held in the US - a testament to the increasingly global scope and impact of TREAT-NMD efforts - from registries, to standards of care, to pre-clinical SOPs, to TACT.  The Conference will be held at the historic Cosmos Club in the DuPont Circle region of DC - a region known for its many embassies, restaurants, and landmarks.  Convenient to the DC Metro subway system, and a short hop to all the free museums on the Washington Mall.


Participation is strictly limited to 200, so sign up early to reserve your spot!



Speakers at the conference include

Arthur Burghes – USA

Kate Bushby – United Kingdom

Annamaria De Luca – Italy

Lawrence Korngut – Canada

Hanns Lochmüller – United Kingdom

Craig McDonald – United States

Lauren Morgenroth – United States

Kanneboyina Nagaraju – United States

Lori Reilly – United States

Volker Straub – United Kingdom

Lee Sweeney – United States

Conference highlights

Biochemical and imaging outcomes

The role of biomarkers in rare neuromuscular diseases for clinical diagnosis or treatment response is becoming more important and their use drives res …

Innovative trial designs and outcome measures

The accelerating pace of clinical development for rare neuromuscular diseases is accompanied by unique sets of challenges and opportunities for all st …

International trial readiness and access to emerging therapies

Increasing numbers of drug development programs in neuromuscular disease put increasing demands on patients, families and the clinical teams, as well …

Post marketing and health economics in orphan disease

Overview With very few drugs available to treat patients with inherited neuromuscular diseases, the main focus of the field has been the move to trial …


Patient registries have proven to be instrumental for clinical research in neuromuscular disorders, and have proven their utility to assess feasibilit …

Standards of care

Therapeutic developments in neuromuscular disorders have highlighted the impact of care and management on the outcomes currently measured in clinical …

The current challenges of preclinical research and study design

In the last years, several treatment approaches for neuromuscular diseases entered clinical trials or pilot trials. The preclinical phase often delive …

The Essential Resource in Achieving Novel Therapies for Neuromuscular Disease: Patient-Focused Drug Development

As we come closer to having therapies for neuromuscular diseases, the issues are becoming increasingly complex.  Whether and how patients and their fa …



Cosmos Club - 2121 Massachusetts Ave NW, Washington, DC 20008, United States