Lysosomal Storage Disorder Industry 2018 : Symptoms, Causes, Types, SWOT Analysis, Size, Share, Growth, Trends and Forecast

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Lysosomal Storage Disorder Market:

Executive Summary

Adroit Helathcare Services’ Lysosomal Storage Disorder Pipeline Analysis – 2018 covers as detailed description of pipeline products that are in various stages of development. The key contents includes the critical clinical end points considered, mechanism of action, expected end of clinical trails, entry into the market and the key companies that are working in the pipeline.

Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. There are nearly 50 of these disorders altogether, and they may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system. New lysosomal storage disorders continue to be identified. While clinical trials are in progress on possible treatments for some of these diseases, there is currently no approved treatment for many lysosomal storage diseases.

Some of the most common lysosomal storage disorders include:

– Gaucher disease: Gaucher disease often causes spleen and liver enlargement, blood problems and bone issues. Learn more about Gaucher disease.

– Fabry disease: This disorder often causes severe burning pains in hands and feet and, in some cases, a distinctive skin rash on the legs. Untreated, this disease can cause kidney failure, heart failure, strokes and death before age 50. Although men are more likely to have severe disease, women may also be seriously affected.

– Niemann-Pick disease: Similar to Gaucher disease, Niemann-Pick disease involves organ enlargement, lung dysfunction and central nervous system damage for certain subtypes.

– Hunter syndrome: This disease is part of a group of disorders that cause bone and joint deformity as well as interference with normal growth.

– Glycogen storage disease II (Pompe disease): Depending on the specific subtype, Pompe disease may cause heart enlargement and heart failure in infants. It may also cause respiratory problems and severe muscle weakness in adults.

– Tay-Sachs disease: This disorder causes severe and fatal mental and physical deterioration, with both an early-onset and a late-onset form.

Companies Mentioned

  • Abeona Therapeutics
  • Actelion Ltd
  • Alexion Pharmaceuticals
  • Amicus Therapeutics
  • AngioChem Inc.
  • ArmaGen
  • Axcentua Pharmaceuticals AB
  • Belrose Pharma, Inc
  • Bioasis Technologies
  • BioMarin Pharmaceutical
  • Bionaturis Group
  • Biosidus
  • BBB Therapeutics
  • Chiesi Group
  • Celltech
  • Cytomedix
  • Dorphan S.A.
  • Etubics Corporation
  • Fate Therapeutics, Inc.
  • Generium
  • Genzyme Corporation
  • Greenovation
  • JCR Pharmaceuticals Co., Ltd.
  • Lixte Biotechnology Holdings, Inc
  • Lysogene

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Table of Content

  • Lysosomal Storage Disorder

o Key updates

o Introduction on Lysosomal Storage Disorder

o Causes

o Epidemiology

o Current treatment landscape

  • Market Analysis

o Market Opportunity Assessment

o Drivers and Barriers

o SWOT analysis of lysosomal Storage Disorder pipeline

o Key players benchmarking

  • Therapeutics under Development by Companies
  • Last Stage Products (Phase III)

o VTS -270 – Mallinckrodt Pharmaceuticals

o Product description

o Clinical trials status

o Clinical Results

o Other development activities

  • Mid Stage Products (Phase II)

o AGT-181 – ArmaGen, Inc.

o Product description

o Clinical trials status

o Clinical results

o Other development activities

  • Early Stage Products (Phase I)

o SB-318 – Sangamo

o Product description

o Clinical trials status

o Clinical Results

o Other development activities

  • Discovery and Pre-clinical stage Products

o ST-920 – Sangamo

o Product description

o Clinical trials status

o Clinical Results

o Other development activities

  • Therapeutic Assessment

o Assessment by Monotherapy Products

o Assessment by Combination Products

o Assessment by Route of Administration

o Assessment by Stage and Route

o Assessment by Molecule Type

o Assessment by Stage and Molecule Type

  • Discontinued Products
  • Competitors Threat Assessment 


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