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Amyotrophic Lateral Sclerosis: A route to larger CNS markets?


Description: Introduction: Amyotrophic lateral sclerosis (ALS) is an orphan neurodegenerative disease which effects less than twenty thousand people across the seven major markets. Nevertheless, ALS is set to provide a platform for drugs for use in more widespread conditions such as Parkinson’s Disease (PD) or Alzheimer’s Disease (AD). Scope: - Epidemiology and prevalence data - Current treatment and unmet market needs - Analysis of trial data, the therapeutic potential of current and future drugs and drug classes - Analysis of key issues in the ALS and orphan disease market supported by key opinion leader interviews Report Highlights: ALS is one of many orphan diseases in the CNS arena. If significant return of investment is to be realized in this market, both existing and future players must address the lack of disease awareness amongst physicians. Currently the only prescription drug approved for ALS is Aventis’ Rilutek (riluzole). Despite the fact that, many physicians doubt its true therapeutic value, Rilutek sales topped $100m in 2002. This demonstrates the scope for commercial opportunities which can be further increased by seeking approval for lucrative indications such as AD or PD. Reasons to Purchase: - Assess the ALS patient potential - Identify the key unmet needs and diagnostic tools, which would highlight novel targets - Review the therapeutic and commercial potential of drugs and compounds in clinical trials based on ALS expert opinion and trial data


Contents: TABLE OF CONTENTS
EXECUTIVE SUMMARY 5
Introduction 5
Scope and coverage of the Brief 5
Key Findings 6
DISEASE OVERVIEW 9
Symptomatology 9
Etiology 10
Prevalence 11
Diagnosis rate 12
Treatment rates 14
Unmet needs 14
TREATMENT 15
Disease-modifying treatments 15
Symptomatic treatments 17
Non-pharmacological treatment 17
R&D PIPELINE 19
Introduction 19
Marketed drugs in clinical trials for ALS 21
Over-the-Counter Drugs for ALS in clinical trials 25
Therapeutic advances and potential market opportunities 26
Pipeline drugs for ALS 27
STRATEGIC OPPORTUNITIES AVAILABLE TO COMPANIES 31
Introduction 31
Strategic opportunites 31
The orphan drug issues in ALS 32
APPENDIX 37
Contributing experts 37
Bibliography 37
Web sites 40
Disclaimer 42





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