Amyotrophic lateral sclerosis

Ra Pharmaceuticals Announces Selection for the First ALS Platform Trial by the Sean M. Healey & AMG Center for ALS at Mass General

Retrieved on: 
Wednesday, September 18, 2019
General Health, Health, Pharmaceutical, Clinical trials, Neurological disorders, Neurodegenerative disorders, Nervous system, Amyotrophic lateral sclerosis, RTT, Sean Healey, ALS Therapy Development Institute, ALS, Zilucoplan, Ra Pharmaceuticals

Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the selection of zilucoplan as one of the first clinical candidates to be evaluated in a pioneering platform trial for amyotrophic lateral sclerosis (ALS), led by the Sean M. Healey & AMG Center for ALS at Mass General.

Key Points: 
  • Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the selection of zilucoplan as one of the first clinical candidates to be evaluated in a pioneering platform trial for amyotrophic lateral sclerosis (ALS), led by the Sean M. Healey & AMG Center for ALS at Mass General.
  • The Healey Center has agreed to provide funding for the execution of the platform trial to support its goal of accelerating the development of effective treatments for patients with ALS.
  • The Healey Center brought together top leaders from around the world to dramatically re-think how we design ALS trials and create a groundbreaking platform trial program, says Merit Cudkowicz, M.D., M.Sc., director of the Sean M. Healey & AMG Center for ALS at Mass General.
  • The ALS platform trial is designed to disrupt the standard pace of ALS therapy development by testing and evaluating multiple treatments simultaneously.

Global Lambert-Eaton Myasthenic Syndrome (LEMS) Market Insights, Epidemiology, and Market Forecast to 2028

Retrieved on: 
Monday, September 16, 2019
Neurological disorders, Organ systems, LEMS, Nervous system, Paraneoplastic syndromes, Myasthenia, Lambert–Eaton myasthenic syndrome, Amyotrophic lateral sclerosis, Catalyst Pharmaceuticals, Myaware

DUBLIN, Sept. 16, 2019 /PRNewswire/ -- The "Lambert-Eaton Myasthenic Syndrome (LEMS) - Market Insights, Epidemiology, and Market Forecast to 2028" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, Sept. 16, 2019 /PRNewswire/ -- The "Lambert-Eaton Myasthenic Syndrome (LEMS) - Market Insights, Epidemiology, and Market Forecast to 2028" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • This report provides the current treatment practices, emerging drugs, market share of the individual therapies, the current and forecasted market size of Lambert-Eaton Myasthenic Syndrome (LEMS) from 2017 to 2028 segmented by seven major markets.
  • The Lambert-Eaton Myasthenic Syndrome (LEMS) market report gives a thorough understanding of the Lambert-Eaton Myasthenic Syndrome (LEMS) by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends.
  • It also provides treatment algorithms and treatment guidelines for Lambert-Eaton Myasthenic Syndrome (LEMS) in the US, Europe, and Japan.

Charles River Laboratories to Host 3rd Annual World Congress

Retrieved on: 
Thursday, September 5, 2019
Research, Medical devices, Biotechnology, Pharmaceutical, Health, General Health, Science, Neurological disorders, Neurodegenerative disorders, Nervous system, Amyotrophic lateral sclerosis, RTT, Les Turner ALS Foundation

Charles River Laboratories International, Inc. is proud to host the 3rd Annual World Congress: Delivering Therapies to the Clinic Faster on September 23-24, 2019 in Cambridge, Mass.

Key Points: 
  • Charles River Laboratories International, Inc. is proud to host the 3rd Annual World Congress: Delivering Therapies to the Clinic Faster on September 23-24, 2019 in Cambridge, Mass.
  • This years World Congress will explore novel approaches to drug development that effectively reduce program timelines and accelerate delivery to the clinic.
  • On September 23, 2019, the World Congress will host a panel to discuss how artificial intelligence (AI) can be applied in drug discovery.
  • Charitable Giving: Charles River will donate 100% of every World Congress registration fee to the Amyotrophic Lateral Sclerosis Therapy Development Institute , a Cambridge-based nonprofit biotech dedicated to finding a cure for ALS.

Emerging Therapeutics for Neurodegenerative Diseases, Part I: Novel Targeted Therapeutic Strategies Technologies for Alzheimer's and Parkinson's Diseases

Retrieved on: 
Thursday, September 5, 2019
Organ systems, Nervous system, Branches of biology, Neuroscience, Neurological disorders, Rare diseases, Neurodegeneration, Senescence, Disease, Amyotrophic lateral sclerosis, Estrogen and neurodegenerative diseases, Neuroimmunology

The most common denominator among neurodegenerative diseases, in addition to nerve cell loss is inflammation.Although, a series of mutant genes and environmental toxins are related to neurodegenerative disorders, the causal mechanisms remain poorly understood.

Key Points: 
  • The most common denominator among neurodegenerative diseases, in addition to nerve cell loss is inflammation.Although, a series of mutant genes and environmental toxins are related to neurodegenerative disorders, the causal mechanisms remain poorly understood.
  • Neurodegenerative diseases can be defined as multifactorial debilitating disorders of the nervous system.
  • Overall, neurodegenerative diseases affect approximately 30 million individuals worldwide, according to the World Health Organization (WHO).
  • Factors Contributing to Neurodegenerative DiseasesThe factors contributing to the onset of neurodegenerative diseases are multiple.

Growth Opportunities in the Neurodegenerative Disorder Therapeutics Market, Forecast to 2024

Retrieved on: 
Thursday, September 5, 2019
RTT, Neurological disorders, Psychiatric diagnosis, Branches of biology, Nervous system, Aging-associated diseases, Geriatrics, Parkinson's disease, Alzheimer's disease, Amyotrophic lateral sclerosis

While companies continue their search, they are also battling declining revenues from marketed products used to manage symptoms.

Key Points: 
  • While companies continue their search, they are also battling declining revenues from marketed products used to manage symptoms.
  • While the therapy market is marred by high rates of pipeline attrition, there are several parallel areas of growth.
  • The study also dives deep into different technological advancements, geographical trends, and potential partnership opportunities for Alzheimer's and Parkinson's diseases.
  • Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

Muscular Dystrophy Association Awards 25 Grants Totaling More Than $6.6 Million for Neuromuscular Disease Research

Retrieved on: 
Thursday, September 5, 2019
Neurological disorders, Organ systems, Muscular dystrophy, Medicine, RTT, Rare diseases, Amyotrophic lateral sclerosis, Muscular Dystrophy Association, Motor neuron diseases, Dystrophy, Congenital muscular dystrophy, Facioscapulohumeral muscular dystrophy

NEW YORK, Sept. 5, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the awarding of 25 new MDA grants totaling more than$6.6 million toward research focused on a variety of neuromuscular diseases (NMDs), including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich's ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy (LGMD), mitochondrial myopathy, myotonic dystrophy (DM), spinal-bulbar muscular atrophy (SBMA), and spinal muscular atrophy (SMA).

Key Points: 
  • NEW YORK, Sept. 5, 2019 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the awarding of 25 new MDA grants totaling more than$6.6 million toward research focused on a variety of neuromuscular diseases (NMDs), including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich's ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy (LGMD), mitochondrial myopathy, myotonic dystrophy (DM), spinal-bulbar muscular atrophy (SBMA), and spinal muscular atrophy (SMA).
  • "MDA's latest group of awards aims to support the most promising research in a variety of neuromuscular diseases," saysLianna Orlando, PhD, interim head of research.
  • The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas.
  • The total list of grant awards for this cycle includes:
    Ludwig Institute for Cancer Research, San Diego
    Ludwig Institute for Cancer Research, San Diego

Neuropore Receives Orphan Drug Designation for NPT520-34 for the Treatment of Amyotrophic Lateral Sclerosis

Retrieved on: 
Wednesday, August 14, 2019
FDA, Clinical trials, Biotechnology, Other Health, Pharmaceutical, Health, General Health, Neurological disorders, Articles, Neurodegenerative disorders, Amyotrophic lateral sclerosis, RTT, Orphan drug, Amyotrophic Lateral Sclerosis, Parkinson’s disease, NPT520-34, Neuropore Therapies, Inc.

Neuropore Therapies, Inc. announced today that it has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS.

Key Points: 
  • Neuropore Therapies, Inc. announced today that it has received orphan drug designation for NPT520-34 for the treatment of amyotrophic lateral sclerosis or ALS.
  • The orphan drug designation will facilitate the development of NPT520-34 in the treatment of this debilitating medical condition for which limited treatments are available.
  • Errol De Souza , President and CEO of Neuropore, stated, We are very pleased with receiving this orphan drug designation from the FDAs Office of Orphan Product Development for NPT520-34 for the treatment of ALS, a devastating disease with a very high unmet medical need.
  • NPT520-34 also has been shown to reduce the expression of markers of neuroinflammation and neuropathology in animal models of amyotrophic lateral sclerosis and Alzheimers disease.

CytRx Corporation Highlights Arimoclomol Licensee Orphazyme A/S Prepares for Regulatory Filing of Arimoclomol in U.S. for Niemann-Pick Disease Type C

Retrieved on: 
Tuesday, July 30, 2019
Neurological disorders, Organ systems, Rare diseases, Medicine, Lipid storage disorders, Inclusion body myositis, Niemann–Pick disease, type C, Niemann–Pick disease, Myositis, Inclusion bodies, Niemann, Amyotrophic lateral sclerosis

As previously announced, Orphazyme intends to file a Marketing Authorization Application (MAA) in Europe in the first half of 2020.

Key Points: 
  • As previously announced, Orphazyme intends to file a Marketing Authorization Application (MAA) in Europe in the first half of 2020.
  • Arimoclomol is in clinical development at Orphazyme for the treatment of Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and amyotrophic lateral sclerosis.
  • Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).
  • Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis.

CytRx Corporation Highlights Arimoclomol Licensee Orphazyme A/S Completes Enrollment in its Phase 3 Clinical Trial of Arimoclomol in Amyotrophic Lateral Sclerosis

Retrieved on: 
Monday, July 22, 2019
Neurological disorders, Rare diseases, Organ systems, Lipid storage disorders, Medicine, Amyotrophic lateral sclerosis, RTT, Niemann–Pick disease, type C, Niemann–Pick disease, Myositis, Draft:BrainStorm Cell Therapeutics

The Phase 3 trial of arimoclomol for ALS is a 76-week, randomized, placebo-controlled trial being conducted at 30 centers of excellence in North America and Europe.

Key Points: 
  • The Phase 3 trial of arimoclomol for ALS is a 76-week, randomized, placebo-controlled trial being conducted at 30 centers of excellence in North America and Europe.
  • Orphazyme anticipates announcing top-line results from the Phase 3 trial in the first half of 2021.
  • We thank Orphazyme for their commitment to the arimoclomol program and their dedication to this patient population."
  • Arimoclomol is in clinical development at Orphazyme for the treatment of Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and amyotrophic lateral sclerosis.

ProMIS Neurosciences Highlights Data for PMN310 at AAIC 2019

Retrieved on: 
Thursday, July 18, 2019
Neurological disorders, Brain, Nervous system, Dementia, Cognitive disorders, Frontal lobe, Temporal lobe, Frontotemporal dementia, Psychiatric diagnosis, Amyotrophic lateral sclerosis, TARDBP, Promis

Additionally, Chief Scientific Officer Dr. Neil Cashman presented data pertaining to the company's preclinical development program selectively targeting toxic forms of TDP43 for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Key Points: 
  • Additionally, Chief Scientific Officer Dr. Neil Cashman presented data pertaining to the company's preclinical development program selectively targeting toxic forms of TDP43 for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
  • On Wednesday, July 17, Dr. Neil Cashman delivered data from ProMIS' preclinical program for ALS.
  • AAIC is the largest, most influential international meeting focused on advancing dementia science.
  • ProMIS is listed on the Toronto Stock Exchange under the symbol PMN, and on the OTCQB Venture Market under the symbol ARFXF.