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

06:26 EDT 26th September 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 [7416 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...

Negative electrospray ionization ion mobility spectrometry combined with paper-based molecular imprinted polymer disks: A novel approach for rapid target screening of trace organic compounds in water samples.

A novel approach for the rapid target screening of water contaminants in trace concentrations was applied for the determination of the artificial sweetener Acesulfame-K, an accepted municipal wastewat...

LC-ESI-MS/MS method for simultaneous determination of eleven bioactive compounds in rat plasma after oral administration of Ling-Gui-Zhu-Gan Decoction and its application to a pharmacokinetics study.

A sensitive and robust LC-MS/MS method has been developed and validated to simultaneous determine the concentrations of tumulosic acid, dehydrotumulosic acid, polyporenic acid C, cinnamic acid, Atract...

Ultrasensitive sensing of Androctonus australis hector scorpion venom toxins in biological fluids using an electrochemical graphene quantum dots/nanobody-based platform.

Rapid and sensitive detection of low levels of scorpion venom toxins in biological fluids is of tremendous importance for decision-taking in cases of envenomation by scorpions stings. In Tunisia, at l...

A chemometric approach for the prediction of the aging levels of automatic transmission fluids by mid-infrared spectroscopy.

Automatic transmission fluids (ATF) are highly complex multi-component systems with a variety of different additive packages which suffer from manifold aging processes due to interfering factors. This...

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...

Ultrasound-assisted vacuum impregnation on the fortification of fresh-cut apple with calcium and black carrot phenolics.

This study evaluated the simultaneous effect of ultrasound on vacuum impregnation process to enhance infusion of calcium lactate and black carrot phenolics into ready to eat apple tissues. A vacuum - ...

Effect of thermoultrasound on aflatoxin M levels, physicochemical and microbiological properties of milk during storage.

The aim of this research was to determine the physicochemical properties, microbial counts and aflatoxin M (AFM) levels of thermoultrasonicated, pasteurized and untreated milk (control) at days 1, 7 a...

Synergistic effect between ultrasound and fierce mechanical activation towards mineral extraction: A case study of ZnO ore.

Though the positive role of ultrasound has been confirmed in the mineral extraction, its potential towards fiercely mechanically-activated mineral was not yet mentioned. In this study, as a novel mech...

News Articles [2209 Associated News Articles listed on BioPortfolio]

The iaedp™ Foundation Announces 2019 Symposium Schedule

The 2019 iaedp™ Symposium, the annual conference renowned as one of the top eating disorders treatment conferences, announced today the Symposium schedule and the opportunity for earning Continuing ...

Lysosomal acid ceramidase ASAH1 controls the transition between invasive and proliferative phenotype in melanoma cells

The contribution of the gut microbiome to neurodevelopment and neuropsychiatric disorders

BOD refrigerators, cell culture products, temperature recorders, cryogenic storage freezers, CO2 incubators

Manufacture,laboratory,refrigerators,freezers,storage,chests,cell,culture,products,temperature,recorders,CO2,incubators

Viking nets $165.2mm via FOPO

Viking Therapeutics Inc. (developing treatments for metabolic and endocrine disorders) netted $165.2mm through a follow-on offering of 9.5mm common shares at $18.50. The company will use the funds for...

Researchers Develop Graphene-based Battery

NewsThe batteries have broad applications in electrified transportation, energy storage, and emergency power supply.

NORD Receives Grant from Anthem Foundation for Expansion of Rare Disease Database®

Washington, D.C., September 25, 2018 – The National Organization for Rare Disorders (NORD) announced today it is enhancing its Rare Disease Database which serves as a valuable source of information ...

ACADIA Pharmaceuticals to Present at the 2018 Cantor Global Healthcare Conference on October 2, 2018

ACADIA Pharmaceuticals Inc. (Nasdaq: ACAD), a biopharmaceutical company focused on the development and commercialization of innovative medicines that address unmet medical needs i...

Two Young Cancer Patients Help Aflac CEO Dan Amos Ring the NYSE Opening Bell

SOURCE: Aflac DESCRIPTION:Earlier this month, two young patients from the Aflac Cancer & Blood Disorders Center at Children's Healthcare of Atlanta joined Aflac executives to ring the opening ...

Video-conferencing brings therapists to patients with anxiety

(Reuters Health) - Talk therapy for anxiety disorders can be effective even when done via video-conference, a new study confirms.

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Companies [510 Associated Companies listed on BioPortfolio]

Alliance for Consumer Education

The Alliance for Consumer Education (ACE) is a 501(c)(3) nonprofit organization founded in 2000 to educate the public about the correct use, storage, and disposal of household and...

Avayle CTS

Avayle CTS, LLC specializes in the global procurement of commercial drugs for clinical trials worldwide. Pharma and biotech companies rely on the Company’s consultative proje...

System1 Biosciences, Inc.

System1 Biosciences, Inc., is a San Francisco-based neurotherapeutics company that employs phenotypic screening to discover novel drugs for complex neurological and psychiatric di...

Stemina Biomarker Discovery Inc.

Stemina Biomarker Discovery was founded in 2007 and is located in Madison, Wisconsin. Stemina is a biomarker discovery company focused on diagnosis and individualized treatment of...

SC Johnson Professional

SC Johnson Professional® is part of SC Johnson®, a family company and one of the world's leading manufacturers of household cleaning products and products for home storage, ...

Ashley Addiction Treatment

Ashley Addiction Treatment (Ashley), celebrating its 35th anniversary this year, is a nationally recognized leader in the integrated, evidence-based treatment of substance-use dis...

Quadrant Biosciences, Inc.

Quadrant Biosciences, Inc. is developing clinical assessment and molecular diagnostic tools to help physicians assess brain health and support more rapid accurate diagnoses of bra...

Onspira Therapeutics

Onspira Therapeutics is a clinical-stage biopharmaceutical company focused on the development of life-changing medicines to bring hope to patients suffering from rare pulmonary di...

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...

Casma Therapeutics, Inc.

Casma Therapeutics is harnessing the natural cellular process of autophagy to open vast new target areas for drug discovery and development. Casma uses several approaches to inter...

Clinical Trials [2870 Associated Clinical Trials listed on BioPortfolio]

Developing a Novel Digital Therapeutic for the Treatment of Generalized Anxiety Disorder

The investigators propose to develop and adapt Unwinding Anxiety phone app specifically for individuals with generalized anxiety disorder.

A Study to Evaluate in Patients With Parkinsonian Type Disorders

A Phase 2 study to evaluate the safety, tolerability and initial efficacy of pramipexole ER, given with aprepitant in patients with parkinsonian type disorders

Mental Health Prevention Among Preschool Children Effectiveness Study

Mental health in Chilean children and families is an urgent public health problem. Prevalence of psychiatric disorders among children between 4 and 11 years old is 27.8%, a higher percenta...

An Online Trauma-informed Yoga Program for Young Adults With Symptoms of Posttraumatic Stress Disorder

Post-traumatic stress disorder (PTSD), a chronic, debilitating condition, is a growing public health concern as the Canadian population has the highest PTSD prevalence worldwide (9.2%; 3.7...

Daytime Bright Light, Circadian Abnormalities, and Delirium in Medical ICU Patients

To determine if daytime bright light will promote circadian alignment and shorten or prevent delirium.

Reducing Self-stigma in Persons With HIV and Drug Use Disorders in Primary Health Care Settings

The investigators propose to lay the groundwork for a larger effectiveness clinical trial that will lead to the advancement of a novel evidence-based treatment for DUD and HIV among Spanis...

The Effect of G-CBT on the Patients With AN : A Randomized Controlled Trial

The study is to examine whether the G-CBT for anorexia nervosa is effective or not. The study will use randomized controlled study design. 80 patients with AN will be recruited from Shangh...

Package of Community-based Mental Healthcare Services in Preventing Perinatal Mental Disorders

During pregnancy and in the year after birth women can be affected by a range of mental health problems. Anxiety and depression are the most prevalent mental illnesses during the perinatal...

Identification of Neurocognitive Disorders by General Practitioners in Primary Care

Neurocognitive disorders (NCD) are today often largely underdiagnosed or diagnosed in late stages in France. Indeed the ALzheimer COoperative Valuation in Europe (ALCOVE) study stated that...

Platelet Antisedimentation Rate and Multiplate Exams in Patients on Antiplatelet Therapy and Controls

To search for simple laboratory methods selecting patients with low/non-responsiveness to P2Y12 receptor antagonists.

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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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