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Lysosomal storage disorders (LSDs) - Biotech, Pharma and Life Science Channel

07:18 EDT 20th June 2018 | BioPortfolio

Lysosomal storage disorders (LSDs) represent a group of about 50 genetic disorders caused by deficiencies of lysosomal proteins. Most involve genetic mutations causing loss of function of key catabolic enzymes residing in the lysosome that are involved in the degradation of macromolecules. The missing lysosomal protein causes a build-up of toxic metabolites in the cells of patients, leading to progressive multisystem disease and premature death. Although individually rare, the combined prevalence of all lysosomal disorders is estimated to be 1 in 8000 births.

Enzyme Replacement Therapy involves restoration of the missing enzyme through periodic intravenous infusion of recombinant enzyme. Exogenously administered lysosomal enzyme re is taken up in cells through cell-surface mannose-6-phosphare receptors, and transported to the lysosome in order to degrade the toxic build-up of accumulated metabolite.

PubMed Articles [7273 Associated PubMed Articles listed on BioPortfolio]

Advances in the discovery of cathepsin K inhibitors on bone resorption.

Cathepsin K (Cat K), highly expressed in osteoclasts, is a cysteine protease member of the cathepsin lysosomal protease family and has been of increasing interest as a target of medicinal chemistry ef...

Pentabromopseudilin: a myosin V inhibitor suppresses TGF-β activity by recruiting the type II TGF-β receptor to lysosomal degradation.

Pentabromopseudilin (PBrP) is a marine antibiotic isolated from the marine bacteria Pseudomonas bromoutilis and Alteromonas luteoviolaceus. PBrP exhibits antimicrobial, anti-tumour, and phytotoxic act...

Biomimetic carbon monoxide delivery based on hemoglobin vesicles ameliorates acute pancreatitis in mice via the regulation of macrophage and neutrophil activity.

Macrophages play a central role in various inflammatory disorders and are broadly divided into two subpopulations, M1 and M2 macrophage. In the healing process in acute inflammatory disorders, shiftin...

Risk factors associated with self-reported musculoskeletal pain among short and long distance industrial gas delivery truck drivers.

This study investigated and compared the associations between self-reported exposures to individual as well as work-related physical and psychosocial risk factors for musculoskeletal (MS) disorders an...

Ultrasound or microwave vacuum thawing of red seabream (Pagrus major) fillets.

Ultrasound assisted vacuum thawing (UVT) or microwave vacuum thawing (MVT) with red seabream fillets were compared to fresh, chill storage thawing, vacuum thawing, microwave thawing and ultrasound tha...

Effect of ultrasonic irradiation on morphology and polymorphic transformation of glycine.

The polymorphic phase transformation of β-glycine to α-glycine was analyzed in the absence and presence of three different intensities of ultrasonic irradiation in a batch system at 25 °C. The ex...

Influence of non-ionic emulsifier type on the stability of cinnamaldehyde nanoemulsions: A comparison of polysorbate 80 and hydrophobically modified inulin.

Cinnamaldehyde nanoemulsions were formulated to enable its application in an aqueous environment. The pure cinnamaldehyde nanoemulsions, stabilized by polysorbate 80 (at concentrations >0.5%), had bot...

Development of a statistical model to detect quality and storage conditions of Atlantic salmon.

The ever-increasing demand for fish as a food, has led to the development of new handling and packaging technologies resulting in premium quality fish products. In order to avoid frauds reaching the m...

Video Synopsis in Complex Situations.

Video synopsis is an effective technique for surveillance video browsing and storage. However, most of the existing video synopsis approaches are not suitable for complex situations, especially crowde...

Yeast mitochondria: an overview of mitochondrial biology and the potential of mitochondrial systems biology.

Mitochondria are dynamic organelles of endosymbiotic origin that are essential components of eukaryal cells. They contain their own genetic machinery, have multicopy genomes and like their bacterial a...

News Articles [1936 Associated News Articles listed on BioPortfolio]

Decibel Therapeutics raises $55m in new funding round

Decibel Therapeutics, a company focused on the discovery and development of medicines to treat hearing loss and hearing-related disorders, has raised $55m in a Series C financing.

EAN Congress 2018: brain disorders – where is the value?

Brain disorders (mental and neurological disorders) represent 35% of Europe’s total disease burden, and a 2010 study showed that the...Read More... The post EAN Congress 2018: brain disorders – wh...

Inflaming sex differences in mood disorders

Atlantic.Net Releases Secure Block Storage (SBS) for its Cloud Servers

ORLANDO, Fla., June 19, 2018/PRNewswire/ --  Atlantic.Net, a leading cloud hostingsolutions provider, today announced the release of Secure Block Storage (SBS), Read more...

Stress disorders tied to increased risk of autoimmune diseases

(Reuters Health) - People who suffer from posttraumatic stress disorder (PTSD) and other stress-related psychiatric issues may be more likely to develop autoimmune diseases than individuals who don't,...

American Psychiatric Nurses Association Calls for End to Immigration Policy that Separates Families

Psychiatric-mental health nursing organization urges national leaders to halt immigration policy. FALLS CHURCH, Va. (PRWEB) June 19, 2018 The American Psychiatric Nurses Association (APNA) calls for ...

Nailing Nail Pathology: Primary Care Case Challenge

Can you 'nail' the diagnosis of these nail disorders? Medscape Family Medicine

Severe Stress May Send Immune System Into Overdrive

TUESDAY, June 19, 2018 -- Trauma or intense stress may up your odds of developing an autoimmune disease, a new study suggests. Comparing more than 106,000 people who had stress disorders with more tha...

Decibel Therapeutics Closes $55 Million Series C Financing

Existing shareholders joined by new institutional investors Decibel Therapeutics, a company dedicated to the discovery and development of breakthrough medicines to trea...

Does Psychological Stress Trigger Autoimmune Disorders?

(MedPage Today) -- F. Perry Wilson, MD, dissects a new study finding a link

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Companies [493 Associated Companies listed on BioPortfolio]

Alcresta Therapeutics, Inc.

Alcresta Therapeutics, Inc. is dedicated to developing and commercializing novel enzyme-based products designed to treat gastrointestinal disorders and rare diseases. The company ...

CleanSlate Centers

A pioneer and leader in outpatient addiction medicine, CleanSlate Centers is a national medical group which provides treatment for the chronic disease of addiction, primarily opio...

Astero Bio Corporation

Astero Bio Corporation is a leader in novel tools and technologies for life science research, pharmaceutical manufacturing, and clinical applications. Astero specializes in the de...

Scioto Biosciences

Based in Indianapolis, Indiana, Scioto Biosciences is a preclinical stage company developing innovative therapies devoted to having a transformative impact on the delivery of micr...

Patientory Stiftung

Patientory Stiftung is a Swiss non-profit membership organization founded to develop and promote new technologies and applications in the healthcare industry, especially ...

Rallybio, LLC

Rallybio was established to identify and accelerate the development of transformative breakthrough therapies for patients with severe and rare disorders. The founders of the compa...

MeiraGTx Limited

MeiraGTx is committed to the development of novel gene therapies to transform the lives of patients suffering from acquired and inherited disorders. The company is developing pote...

Caron Treatment Centers

Caron Treatment Centers is a nationally recognized non-profit provider of alcohol and drug addiction treatment. With more than 50 years in the field, Caron is one of the oldest and largest facilities ...

Exicure, Inc.

Exicure, Inc. is a clinical stage biotechnology company developing a new class of immunomodulatory and gene silencing drugs against validated targets. Exicure’s 3-dimensional...

Rotech Healthcare Inc.

Rotech Healthcare Inc. is one of the largest providers of home medical equipment and related products and services in the United States, with a comprehensive offering of respirato...

Clinical Trials [2703 Associated Clinical Trials listed on BioPortfolio]

DBS in the Treatment of Intractable Movement Disorders

The aim of this study is to observe the efficacy of Deep Brain Stimulation in the treatment of Parkinson's disease,Essential Tremors and Dystonia in our locality.

Swallowing Disorders in Minimally Consciousness Patients (MCS)

This study aimed to characterize swallowing disorders in minimally consciousness patients after brain traumatic injury.

Implementing and Sustaining a Transdiagnostic Sleep and Circadian Treatment

The goal is to collect pilot data on an adapted version of the Transdiagnostic Sleep and Circadian intervention (TranS-C), referred to as 'Fitted TranS-C'. We seek to determine if Fitted T...

Furosemide for Accelerated Recovery of Blood Pressure Postpartum: a Randomized Controlled Trial

A randomized, double-blind, placebo-controlled single center investigation of furosemide's effect on postpartum blood pressure control in pregnancies affected by hypertensive disorders of ...

Monitoring of Immune and Microbial Reconstitution in (HCT) and Novel Immunotherapies

This protocol serves as a mechanism to collect, store, and distribute bodily fluid and tissue samples obtained from Hematopoietic Cell Transplant (HCT) or novel immunotherapy patients and ...

Serious Games Rehabilitation Programme to Treat Gait and Balance Disorders in PD Patients

To test the effects of a serious game rehabilitation programme in 50 patients with Parkinson's disease with gait and balance disorders in a randomised controlled study

Implementation and Evaluation of SBIRT

Alarming rates of unhealthy alcohol, non-prescription drug, and tobacco use highlight the preventable health risks of substance abuse and the urgent need to advance behavioral health syste...

Clinical Decision Support for Opioid Use Disorders in Medical Settings: Usability Testing in an EMR

The primary objective of this pilot study is to program an opioid use disorder (OUD) clinical decision support (CDS) tool for use in an electronic medical record (EMR) and obtain high prim...

Postural Garment Versus Exercises for Women With Cervical Pain

This study aim to compare a new postural garment (Posture Plus Force ®) versus exercises in women with non specific cervical pain. The investigators focus on nurses and allied health prof...

Study of Hemostasis in Patients With Congenital Disorder of Glycosylation

The purpose of this study is to investigate the coagulation balance in a cohort of congenital disorder of glycosylation (CDG) patients using conventional tests combined with an integrated ...

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Medical and Biotech [MESH] Definitions

Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.

An abundant lysosomal-associated membrane protein that has been found to shuttle between LYSOSOMES; ENDOSOMES; and the PLASMA MEMBRANE. Loss of expression of lysosomal-associated membrane protein 2 is associated with GLYCOGEN STORAGE DISEASE TYPE IIB.

A group of enzymatic disorders affecting the nervous system and to a variable degree the skeletal system, lymphoreticular system, and other organs. The conditions are marked by an abnormal accumulation of catabolic material within lysosomes.

A group of autosomal recessive lysosomal storage disorders marked by the accumulation of GANGLIOSIDES. They are caused by impaired enzymes or defective cofactors required for normal ganglioside degradation in the LYSOSOMES. Gangliosidoses are classified by the specific ganglioside accumulated in the defective degradation pathway.

The severe infantile form of inherited lysosomal lipid storage diseases due to deficiency of acid lipase (STEROL ESTERASE). It is characterized by the accumulation of neutral lipids, particularly CHOLESTEROL ESTERS in leukocytes, fibroblasts, and hepatocytes. It is also known as Wolman's xanthomatosis and is an allelic variant of CHOLESTEROL ESTER STORAGE DISEASE.

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