Orphan Drugs for CNS Diseases: R&D and Market 2014-2024

NEW YORK, March 3, 2014 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

Orphan Drugs for CNS Diseases: R&D and Market 2014-2024

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Report Details

Uncommon nerve disorders - new study explains technological and commercial prospects

What's the future of medicines for rare central nervous system (CNS) disorders? Visiongain's new report gives you predictions for those therapies. Stay ahead. There you explore sales results, technologies, R&D trends, opportunities, and revenue forecasts.

So see what's possible for treating unusual neurological conditions. Avoid missing out. You gain data and financial analysis for that industry and market, appraising its outlooks. Find technological progress and commercial prospects, seeing potential gains.

Read on to explore those treatments and find what their future could be worth.

Forecasts and other analyses for therapy of rare (orphan) neurodegenerative disorders

Besides forecasting revenues to 2024, that work shows historical results, growth rates, and market shares. There you get feel for technologies, regulatory outlooks, and developments (R&D). You also gain 41 tables, 40 charts, and two interviews with the industry.

Knowledge to help your work and save time, helping your influence

Our report strives to help your business research, analyses, and decisions on treatments for debilitating CNS diseases. There also see how you can save time. And find how you could benefit your authority through better understanding the present and future.

So the following sections explain what you get in that new investigation.

Forecasting of that world market and its main segments - you explore possibilities

Also in that report you see individual revenue predictions to 2024 for four therapeutic submarkets at world level:• Huntington's disease (HD)• Amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease, motor neurone disease)• Myasthenia gravis (MG)• Other treatment applications.

How will those categories expand? Which classes of therapy can generate most money? So you assess outlooks for sales growth, seeing where you can gain.

There you explore competition and rising sales for treating the rarer abnormalities causing neurodegeneration, autoimmune neuromuscular disorder, and other nerve disease.

You also assess top products' futures.

Predictions of leading agents' revenues

Find sales outlooks for brands, too, seeing how they compete and succeed. Our study shows you individual revenue forecasts to 2024 for four marketed drugs:

• Xenazine (tetrabenazine)

• Rilutek (riluzole)

• Mestinon (pyridostigmine)

• Imuran (azathioprine).

There you find products and years with highest predicted sales and growth. Also you examine competitors. You see what's happening, then, understanding challenges, competition, opportunities, and potentials.

Our study also divides its overall prediction into geographical regions.

Healthcare in leading national markets - what outlooks for those CNS drug sales?

Our analyses show you individual revenue forecasts to 2024 for 11 national markets:• United States (US)• Japan• EU leaders - Germany, France, the UK, Italy, and Spain (EU5)• BRIC nations - Brazil, Russia, India, and China.

There you find countries with highest revenues, demand, and potential sales growth. Our work explains. You assess needs and demands for those specialist medicines.

Explore, too, how that neurological pharma technology benefits developers, producers, marketers, and sellers. Our work shows you, discussing issues.

Events and forces for developing, producing, marketing, and selling CNS orphan medicines

Our report shows you issues and events affecting that pharmaceuticals sector from 2014, including these:• Candidates in development - R&D pipelines - for those indications• Benefits of orphan drug designation• Treatment needs - unmet and under-met - stimulating demand• Regulatory incentives for developers, producers, and sellers.

And these influences:

• Drug launches expected and their implications for that industry and market

• Potential for orphan CNS products in emerging national markets

• Innovations - including drug-device combinations, neurotrophic factors to reverse disease, cellular therapy, and stimulating of neuronal growth

• Companion diagnostics and personalised medicine.

There you explore political, economic, social, and technological questions, investigating outlooks for commerce. Discover what the future holds.

Examine, then, what stimulates and restrains players in that industry, and affects results.

Companies and 2018 market value - find prospects for success

Our report, then, predicts the world market for drugs treating those rare CNS conditions will exceed $2.5bn in 2018. There big pharma and specialty companies can gain. Explore how.

There you examine activities of many companies, including these:

• GlaxoSmithKline (GSK)

• Lundbeck

• Valeant Pharmaceuticals

• Teva Pharmaceutical Industries

• BrainStorm Cell Therapeutics.

R&D in that industry holds strengths, and from 2014 there will arise many opportunities. From this decade, patients, payers, and pharmaceutical companies will benefit.

And you discover how that market can perform, staying ahead in knowledge.

Ways Orphan Drugs for CNS Diseases: R&D and Market 2014-2024 helps you

• Revenues there to 2024 at world level, for 4 submarkets, and 4 top products - examine outlooks for production, marketing, and sales

• Forecasts to 2024 for 11 national markets in the Americas, Europe, and Asia - assess developed and developing countries for potential revenues

• Prospects for established competitors, emerging companies, and new entrants - explore portfolios, results, strategies, and developmental activities.

Information found nowhere else, helping your searches, analyses, and plans

With that data you're less likely to fall behind in knowledge or miss opportunity. Being free to choose, you find how you could save time and effort with our study, helping your decisions.

Table of Contents

1.1 Orphan Drugs for CNS Diseases: Market Overview

1.2 Report Contents - Chapter Overviews

1.3 Research and Analysis Methods

2. Introduction to Orphan Drugs for CNS Disorders

2.2 What are the Major Orphan CNS Indications?

2.2.1 Huntington's Disease

2.2.2 Amyotrophic Lateral Sclerosis (ALS)

2.2.3 Myasthenia Gravis

2.2.4 Other Orphan CNS Indications

2.3 Orphan Drug Designation: Providing Incentives to Companies

2.3.1 What Benefits Come with Orphan Drug Designation?

2.4 Demand for Orphan Drugs Gathers Momentum

2.4.1 Development of Orphan Drugs

3. The World Orphan CNS Drugs Market, 2014-2024

3.1.1 Huntington's Disease: The Largest Orphan CNS Disease Sector in 2012

3.2 The Orphan CNS Drug Market: Revenue Forecast, 2013-2024

3.2.1 What Will Drive Growth in the Market to 2024?

3.2.2 What Will Restrain the Market From 2014-2024?

3.3 Orphan Drugs in Huntington's Disease

3.3.1 Huntington's Disease: Submarket Forecast, 2013-2024

3.3.1.1 Huge Unmet Need to Drive HD Treatment Market

3.3.1.2 Enactment of New Law in US May Add Growth Impetus

3.4 Orphan Drugs in Amyotrophic Lateral Sclerosis

3.4.1 Amyotrophic Lateral Sclerosis: Submarket Forecast, 2013-2024

3.4.1.1 New Drug Launches Will Provide Market Recovery Following Patent Expiry of Rilutek

3.5 Orphan Drugs in Myasthenic Gravis

3.5.1 Myasthenia Gravis: Submarket Forecast, 2013-2024

3.5.1.1 Clinical R&D into New Treatments Keep Patients Hopeful

4. Leading National Markets for Rare CNS Indications 2014-2024

4.2 Leading National Markets: Revenue Forecasts, 2013-2024

4.3 The US: The Leading Market for Orphan CNS Drugs in 2012

4.3.1 Orphan Drug Regulations in the US

4.3.2 Benefits for Orphan Drug Developers

4.3.3 The US Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.4 Orphan Drugs in Leading EU Markets

4.4.1 The EU5 Orphan CNS Drug Market: Revenue Forecasting, 2013-2024

4.4.1.1 The Incentives for Orphan Drug Development in the EU

4.4.2 Orphan CNS Drugs in the EU5: Submarket Revenue Forecasts, 2013-2024

4.4.2.1 The German Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.4.2.2 The French Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.4.2.3 The UK Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.4.2.4 The Spanish Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.4.2.5 The Italian Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.5 The Japanese Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.5.1 Orphan Drug Regulations and Benefits in Japan

4.6 Potential for Orphan CNS Drugs in Emerging National Markets, 2013-2024

4.6.1 The Chinese Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.6.2 The Indian Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.6.2.1 Large Patient Population to Draw Drug Developers to India

4.6.3 The Russian Orphan CNS Drug Market: Revenue Forecast, 2013-2024

4.6.3.1 Attempts to Introduce Orphan Designation and Incentives 2010-2013

4.6.4 The Brazilian Orphan CNS Drug Market: Revenue Forecast, 2013-2024

5. Outlooks for Leading Orphan CNS Drugs 2014-2024

Huntington's

5.1.1 Xenazine (Tetrabenazine; Lundbeck/Valeant Pharmaceuticals)

5.1.1.1 Omerus Pipeline HD Treatment Receives Orphan Designation

5.1.1.2 Xenazine: Revenue Forecast, 2013-2024

5.2 Amyotrophic Lateral Sclerosis Treatments

5.2.1 Rilutek (Riluzole; Covis Pharmaceuticals)

5.2.1.1 Patent Expiry to Cause Influx of Generic Treatments

5.2.1.2 Rilutek: Revenue Forecast, 2013-2024

5.3 Myasthenia Gravis Treatments

5.3.1 Mestinon (Pyridostigmine bromide; Valeant Pharmaceuticals)

5.3.1.1 Lack of Novel Treatments to Compete With Branded Drug

5.3.1.2 Mestinon: Revenue Forecast, 2013-2024

5.3.2 Imuran (Azathioprine; Prometheus Laboratories/Aspen Pharmacare)

5.3.2.1 Serious Adverse Events May Further Reduce Use of Imuran

5.3.2.2 Imuran: Revenue Forecast, 2013-2024

6. Orphan CNS Drugs: R&D Pipelines

Huntington's

6.1.1 ALN-HTT (Alnylam Pharmaceuticals/Medtronic Inc.): Drug-Device Combination Therapy

6.1.2 HD-02 (Avicena Group Inc.): Correcting the Body's Cellular Energy

6.1.3 CERE-120 (Ceregene Inc.): Delivering Neurotrophic Factors to Reverse Disease Process

6.1.4 Lead Compounds (Chaperone Therapeutics): Targeting Protein Misfolding

6.1.5 CX929 (Cortex Pharmaceuticals): Ampakine Treatment to Encourage Neuronal Growth

6.1.6 EPI-743 (Edison Pharmaceuticals): Redox Therapy in Preclinical Testing

6.1.7 GSK356278 (GSK): Respiration Treatment in Line for Additional HD Indication

6.1.8 Modified Antisense Oligo-Nucleotides (Isis Pharmaceuticals): Partnering with Roche to Aid Drug Discovery

6.1.9 NsG33 (Meteorin; NsGene A/S): Neurotrophic Protein Shown to Stimulate Neurite Growth in Preclinical Studies

6.1.10 LSD-1/MAO-B Inhibitor (Oryzon Genomics): Reducing Enzyme Activity May Provide Therapeutic Benefit

6.1.11 PBT2 (Prana Biotechnology): Targeting Metal-Protein Interactions to Reduce Protein Aggregation

6.1.12 PRO289 (Prosensa): Lowering Levels of Mutant Gene Proteins

6.1.13 RP103 (Raptor Pharmaceutical Corporation): Cysteamine Treatment Receives Orphan Designation

6.1.14 Zinc DNA Transcription Factors (Sangamo Biosciences/Shire AG): Switching Genes On and Off

6.1.15 Selisistat (SEN196; Siena Biotechnology): Orphan Drug Targeting Sirtuin Protein Inhibition in Phase II Development

6.1.16 Huntexil (Teva Pharmaceuticals): Neutralizing Dopaminergic Activity

6.2 Drugs in the Amyotrophic Lateral Sclerosis Pipeline, 2013

6.2.1 ALS-02, ALS-05 and ALS-08 (The Avicena Group): Designated Orphan Drug in Advanced Clinical Testing

6.2.2 NurOwn™ (BrainStorm Cell Therapeutics): Stem Cell Treatment to Replace Damaged Cells

6.2.3 Tirasemtiv (CK-2017357; Cytokinetics): Skeletal Muscle Activator Advances to Phase II Trials

6.2.4 E0302 (Mecobalamin; Eisai): Vitamin B12 Therapy Shown to Improve Muscle Wasting

6.2.5 Ozanezumab (GSK1223249; GSK): Monoclonal Antibody Treatment Recruiting Patients for Phase II Trial

6.2.6 Radicut (Edaravone; Mitsubishi Tanabe): Free Radical Scavenger in Advanced Trials

6.2.7 Region Specific Stem Cells (Neuralstem): Neuronal Stem Cell Therapy in Preclinical Testing

6.2.8 NP001 (Neuraltus Pharmaceuticals): Altering Macrophage Activity

6.2.9 sNN0029 (NeuroNova): Growth Factor Treatment Shown to Improve Muscle Strength

6.2.10 TDI-132 (Gilenya™; Novartis & The ALS Therapy Development Institute): Therapy to Counteract Aggressive Immune Cells

6.3 Drugs in the Myasthenia Gravis Pipeline, 2013

6.3.1 Soliris® (Eculizumab; Alexion Pharmaceuticals): Blood Disorder Treatment Investigated as Potential MG Therapy

6.3.2 BHT-3034 (Bayhill Therapeutics): Vaccine Therapy to Reverse Harmful Immune Responses

6.3.3 BKT130 (Biokine Therapeutics): Chemokine Inhibitor

6.3.4 CK-2017357 (Tirasemtiv; Cytokinetics): Designated Orphan Drug Tested in Multiple Indications

7. Orphan Drugs in CNS: Industry Trends, 2014-2024

7.2 Orphan Drugs in CNS: Opportunities and Threats, 2013-2024

7.3 Orphan Drugs in CNS: STEP Analysis, 2013-2024

7.3.1 Social Factors

7.3.1.1 Ethical Considerations in Funding Orphan Drug Development

7.3.2 Technological Developments

7.3.2.1 Increased Understanding of Disease Will Drive Innovation

7.3.3 Economic Pressures

7.3.3.1 Discounts in Drug Prices Will Increase Accessibility

7.3.4 Political Issues

7.3.4.1 Harmonising Global Regulatory Pathways

7.4 Trends in Orphan CNS Drug Development

7.4.1 Clinical Trials for Orphan Drugs

7.4.1.1 Are Adverse Events More Acceptable for Orphan Drugs?

7.4.1.2 Challenges and Opportunities in Patient Recruitment

7.4.2 Biomarkers for Orphan CNS Indications

7.4.2.1 Companion Diagnostics: Driving Personalised Medicine

7.4.3 The Role of Public Funding in Orphan Drug Development

7.5 Commercialising Orphan Drugs in CNS

7.5.1 Pricing of Orphan Drugs May Become Crucial to Success

7.5.2 Market and Patient Access Are Crucial for Orphan Drug Success

7.5.3 Repositioning Drugs for Orphan CNS Indications

7.5.3.1 Expanding Beyond Orphan Indications

7.5.4 Big Pharma and Orphan Drugs

7.5.4.1 Orphan Drugs Fit the Personalised Medicine Model

7.5.5 Common Market Entry Strategies

7.5.6 Orphan Indications as an Opportunity for Generic Drug Manufacturers

8. Research Interviews

8.1.1 Avicena Group

8.1.2 Rare Diseases and Applying for Orphan Drug Status

8.1.3 HD-02: Targeting the Body's Cellular Energy Systems

8.1.4 Challenges in Developing Orphan Drugs

8.1.5 Future Growth Opportunities and Perspective for the Market

8.2 Respondent from Edison Pharmaceuticals

8.2.1 Edison Pharmaceuticals

8.2.2 Defining Orphan Disease and Drug Submissions in Target Markets

8.2.3 The Market Potential (and Risk) of Pursuing Treatments for Mitochondrial Diseases

8.2.4 Company Alliances and Future Prospects for the Market

9. Conclusions from Our Study

9.2 Renewed Interest in Rare CNS Diseases will Stimulate that Market

9.3 The US will Maintain its Market Dominance from 2014-2024

9.4 High Price of Treatments May Limit Market Potential

List of Tables

Table 1.2 Common Abbreviations Used in this Report

Table 2.1 EU Prevalence Statistics for Selected Rare CNS Indications, 2013

Table 3.1 Global Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Sector, 2012

Table 3.2 Global Orphan CNS Drug Market: Overall Market and Revenue Forecasts by Sector ($m, AGR %, CAGR %), 2013-2024

Table 3.3 Global Orphan CNS Drug Market: Submarket Shares (%), 2012-2024

Table 3.4 Huntington's Disease Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 3.5 Amyotrophic Lateral Sclerosis Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 3.6 Myasthenia Gravis Submarket: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.1 Global Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Region, 2012

Table 4.2 Global Orphan CNS Drug Market: Revenue Forecasts ($m, AGR %, CAGR %) by Region, 2013-2024

Table 4.3 Global Orphan CNS Drug Market: Regional Submarket Shares (%),2012-2024

Table 4.4 US Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.5 EU5 Orphan CNS Drug Market: Revenue ($m) and Market Share (%) by Country, 2012

Table 4.6 EU5 Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %) by Country, 2013-2024

Table 4.7 Fee Reductions (%) Available for SMEs Developing Orphan Drugs in the EU, 2013

Table 4.8 German Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.9 French Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.10 UK Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.11 Spanish Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.12 Italian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.13 Japanese Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.14 Chinese Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.15 Indian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.16 Russian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 4.17 Brazilian Orphan CNS Drug Market: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 5.1 Top Orphan CNS Drugs: Revenues ($m) and Leading Revenue Shares (%), 2012

Table 5.2 Xenazine: Revenue ($m), 2009-2012

Table 5.3 Xenazine: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 5.4 Rilutek: Revenue ($m), 2010-2012

Table 5.5 Rilutek: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 5.6 Mestinon: Revenue ($m), 2010-2012

Table 5.7 Mestinon: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 5.8 Imuran: Revenue ($m), 2008-2012

Table 5.9 Imuran: Revenue Forecast ($m, AGR %, CAGR %), 2013-2024

Table 6.1 Selected Pipeline Candidates in Development for Huntington's Disease, 2012

Table 6.2 Selected Pipeline Candidates in Development for ALS, 2013

Table 6.3 Selected Pipeline Candidates in Development for Myasthenia Gravis, 2013

Table 7.1 Global Orphan CNS Drug Market: Strengths and Weaknesses, 2013-2024

Table 7.2 Global Orphan CNS Drug Market: Opportunities and Threats, 2013-2024

Table 9.1 Global Orphan CNS Drug Market: Revenue ($m, CAGR %), and Market Share (%) by Sector, 2012, 2018 & 2024

Table 9.2 Global Orphan CNS Drug Market: Revenue ($m, CAGR %) and Market Share (%) by Region, 2012, 2018 & 2024

List of Figures

Figure 2.2 Orphan Drugs Clinical Pipeline by Therapeutic Category, 2012

Figure 2.3 Orphan Drug Designation Benefits in Major Markets, 2013

Figure 3.1 Orphan Drug Approvals in the US, 2000-2013

Figure 3.2 Global Orphan CNS Drug Market: Share (%) by Sector, 2012

Figure 3.3 Global Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 3.4 Global Orphan CNS Drug Market: Submarket Shares (%), 2018

Figure 3.5 Global Orphan CNS Drug Market: Submarket Shares (%), 2024

Figure 3.6 Global Orphan CNS Drug Market: Drivers and Restraints, 2014-2024

Figure 3.7 Huntington's Disease Market: Revenue Forecast ($m), 2013-2024

Figure 3.8 Amyotrophic Lateral Sclerosis Market: Revenue Forecast ($m), 2013-2024

Figure 3.9 Myasthenia Gravis Market: Revenue Forecast ($m), 2013-2024

Figure 4.1 Global Orphan CNS Drug Market: Share (%) by Region, 2012

Figure 4.2 Global Orphan CNS Drug Market: Share (%) by Region, 2018

Figure 4.3 Global Orphan CNS Drug Market: Share (%) by Region, 2024

Figure 4.4 US Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.5 EU5 Orphan CNS Drug Market: Share (%) by Country, 2012

Figure 4.6 EU5 Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.7 German Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.8 French Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.9 UK Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.10 Spanish Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.11 Italian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.12 Japanese Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.13 Chinese Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.14 Indian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.15 Russian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 4.16 Brazilian Orphan CNS Drug Market: Revenue Forecast ($m), 2013-2024

Figure 5.1 Top Orphan CNS Drug: Revenue Shares (%), 2012

Figure 5.2 Xenazine: Revenue ($m), 2009-2012

Figure 5.3 Xenazine: Revenue Share (%) by Region, 2012

Figure 5.4 Xenazine: Revenue Forecast ($m), 2013-2024

Figure 5.5 Rilutek: Revenue ($m), 2010-2012

Figure 5.6 Rilutek: Revenue Forecast ($m), 2013-2024

Figure 5.7 Mestinon: Revenue ($m), 2010-2012

Figure 5.8 Mestinon: Revenue Forecast ($m), 2013-2024

Figure 5.9 Imuran: Revenue ($m), 2008-2012

Figure 5.10 Imuran: Revenue Forecast ($m), 2013-2024

Figure 9.1 Global Orphan CNS Drug Market: Revenue ($m) by Sector, 2012, 2018 & 2024

Figure 9.2 Global Orphan CNS Drug Market: Revenue ($m) by Region, 2012, 2018 & 2024

Companies Listed

Agência Nacional de Vigilância Sanitária (ANVISA, Brazil)

Alnylam Pharmaceuticals

Apotex

Aspen Pharmacare

Avicena Group

Bayhill Therapeutics

BHR Pharma

Biogen Idec

Biokine Therapeutics

Biovail

BrainStorm Cell Therapeutics

California's Stem Cell Agency

Cambridge Laboratories

Ceregene

Chaperone Therapeutics

Committee for Orphan Medicinal Products (COMP)

Core Pharmaceuticals

Cortex Pharmaceuticals

Covis Pharmaceuticals

Cytokinetics

Edison Pharmaceuticals

Eisai

European Commission

European Medicines Agency (EMA)

European Union

Food and Drug Administration (FDA, US)

Glenmark Generics

Global Pharmaceuticals (generics division of Impax Laboratories

GlaxoSmithKline (GSK)

Huntington's Disease Society of America (HDSA)

Isis Pharmaceuticals

Lundbeck

Massachusetts General Hospital

Medtronic

Ministry of Health, Labor and Welfare (MHLW, Japan)

Mitsubishi Tanabe

National Institute for Health and Care Excellence (NICE)

National Institute for Neurological Disorders and Stroke (NINDS)

Neuralstem

Neuraltus Pharmaceuticals

NeuroNova

NeuroSearch

Novartis

NsGene

Omerus Corporation

Oryzon Genomics

Pfizer

Prana Biotechnology

Prestwick Pharmaceuticals

Prometheus Laboratories

Prosensa

Raptor Pharmaceuticals

Rare Disease UK

Roche

Roxane Pharmaceuticals

Russian Ministry of Health and Social Development (MOHSD)

Sangamo Biosciences

Sanofi

Shire

Siena Biotechnology

State Food and Drug Administration (SFDA, China)

Sun Pharmaceutical Industries

Teva Pharmaceuticals

The ALS Therapy Development Institute

University Hospital Tübingen

University of Kyoto

Valeant Pharmaceuticals

World Health Organisation (WHO)

Zydus Pharmaceuticals

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