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!

 

Annemieke
Eric

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 …



Registries

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 …



Location

 

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

www.cosmosclub.org