Motor neuron diseases

Metabolon and Sheffield Institute for Translational Neuroscience Join Forces to Propel ALS Research and Biomarker Discovery

Retrieved on: 
Tuesday, January 23, 2024
Patient, MNDA, Research, MND, Partnership, Metabolomics, CSF, Translational neuroscience, University, Brain cell, Medicine, Motor neuron diseases, Cell, Plasma, Disease, Facial muscles, Fluid mechanics, Biomarker, Neurology, Nervous system, Metabolism, Diagnosis, Motor Neurone Disease Association, Pharmaceutical industry

MORRISVILLE, N.C., Jan. 23, 2024 /PRNewswire/ -- Metabolon, Inc., the global leader in providing metabolomics solutions advancing a wide variety of life science research, diagnostic, therapeutic development, and precision medicine applications, today announced a strategic partnership with the Sheffield Institute for Translational Neuroscience (SITraN) and A Multicentre Biomarker Resource Strategy in ALS (AMBRoSIA). The collaboration aims to explore novel pathogenic mechanisms of ALS that may expand therapeutic possibilities to a patient population in need of additional treatment options.

Key Points: 
  • Motor neuron diseases (MND), which include ALS, are progressive disorders that affect voluntary muscle control.
  • Currently, there is no cure for these diseases, and approximately 80% of people with ALS die within two to five years of diagnosis.
  • "Metabolon is thrilled to collaborate with SITraN, a leading institution in translational neuroscience, and AMBRoSIA.
  • Together, we aim to drive significant progress in ALS research and biomarker discovery, bringing us closer to effective diagnostic and therapeutic solutions for this challenging disease," said Karl Bradshaw, Chief Business Officer at Metabolon.

Metabolon and Sheffield Institute for Translational Neuroscience Join Forces to Propel ALS Research and Biomarker Discovery

Retrieved on: 
Tuesday, January 23, 2024
Patient, MNDA, Research, MND, Partnership, Metabolomics, CSF, Translational neuroscience, University, Brain cell, Medicine, Motor neuron diseases, Cell, Plasma, Disease, Facial muscles, Fluid mechanics, Biomarker, Neurology, Nervous system, Metabolism, Diagnosis, Motor Neurone Disease Association, Pharmaceutical industry

MORRISVILLE, N.C., Jan. 23, 2024 /PRNewswire/ -- Metabolon, Inc., the global leader in providing metabolomics solutions advancing a wide variety of life science research, diagnostic, therapeutic development, and precision medicine applications, today announced a strategic partnership with the Sheffield Institute for Translational Neuroscience (SITraN) and A Multicentre Biomarker Resource Strategy in ALS (AMBRoSIA). The collaboration aims to explore novel pathogenic mechanisms of ALS that may expand therapeutic possibilities to a patient population in need of additional treatment options.

Key Points: 
  • Motor neuron diseases (MND), which include ALS, are progressive disorders that affect voluntary muscle control.
  • Currently, there is no cure for these diseases, and approximately 80% of people with ALS die within two to five years of diagnosis.
  • "Metabolon is thrilled to collaborate with SITraN, a leading institution in translational neuroscience, and AMBRoSIA.
  • Together, we aim to drive significant progress in ALS research and biomarker discovery, bringing us closer to effective diagnostic and therapeutic solutions for this challenging disease," said Karl Bradshaw, Chief Business Officer at Metabolon.

bit.bio further expands ioCells™️ range with early access launch of ioMotor Neurons™️ for research and drug discovery in diseases such as amyotrophic lateral sclerosis (ALS)

Retrieved on: 
Wednesday, December 6, 2023
Health, Stem Cells, Genetics, Research, Science, Pharmaceutical, Biotechnology, CNS, ALS, Motor neuron diseases, Toxicology, Patient, Multimedia, Culture, Weakness, Automation, Motor neuron, Drug discovery, Neuron, Cell, CRISPR, Death, Brain cell, Innovation, Aroma compound, Pharmaceutical industry, Medical device

This takes the total number of products in the ioCells range - human cells for research and drug discovery - to 20 products, including wild type, CRISPR-ready and disease model cells.

Key Points: 
  • This takes the total number of products in the ioCells range - human cells for research and drug discovery - to 20 products, including wild type, CRISPR-ready and disease model cells.
  • The launch of ioMotor Neurons also follows the recent opening of bit.bio’s new manufacturing and automation laboratories by the UK’s Minister for Science, Research and Innovation.
  • Damage or death of motor neurons can lead to a devastating group of conditions called Motor Neuron Diseases (MNDs).
  • ioMotor Neurons are an important part of our ALS toolkit that can now be incorporated into motor neuron disease research and early drug discovery workflows in ways that were not previously possible.

NeuroSense Granted Patents in Europe, Japan, and Israel for its ALS Drug PrimeC

Retrieved on: 
Tuesday, September 19, 2023
Maintenance, Israel Patent Office, Food and Drug Administration, Celecoxib, IP, Motor neuron diseases, Patent, Mood stabilizer, Ciprofloxacin, FDA, Food, Pharmaceutical industry, ALS

The patents relate to NeuroSense's unique fixed-dose combination of ciprofloxacin and celecoxib, two U.S. Food and Drug Administration (FDA) approved drugs that are the active ingredients in PrimeC, the Company's lead drug candidate for treating Amyotrophic Lateral Sclerosis (ALS).

Key Points: 
  • The patents relate to NeuroSense's unique fixed-dose combination of ciprofloxacin and celecoxib, two U.S. Food and Drug Administration (FDA) approved drugs that are the active ingredients in PrimeC, the Company's lead drug candidate for treating Amyotrophic Lateral Sclerosis (ALS).
  • Corresponding patents have been issued in the U.S., Canada, and Australia and are valid through 2038, subject to appropriate maintenance.
  • Additional patent applications relating to the novel formulation of PrimeC and methods for the treatment of other neurodegenerative diseases using NeuroSense's platform of combination therapies including StabiliC and CogniC are pending as well.
  • ALS remains a pressing unmet need, and our unwavering commitment to advancing our program, supported by robust intellectual property, underscores our dedication to delivering an effective therapy for individuals battling ALS."

SadBaby is Opening New Doorways for Providing Financial Aid to SMA Patients

Retrieved on: 
Thursday, July 29, 2021
Neurological disorders, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Spinal muscular atrophy, SMA

ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.

Key Points: 
  • ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.
  • The SadBaby Team aims to improve every patient's quality of life and gather sufficient funds to support the affected children and their families.
  • Working together with SMA Association in Antalya, SadBaby has already raised significant donations for babies affected with SMA and is soon organising a concert to further their funding strategy.
  • SadBaby is also planning to create an NFT marketplace where families would be able to sell NFT products and obtain financial help by this year's end.

SadBaby is Opening New Doorways for Providing Financial Aid to SMA Patients

Retrieved on: 
Thursday, July 29, 2021
Neurological disorders, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Spinal muscular atrophy, SMA

ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.

Key Points: 
  • ANTALYA, Turkey, July 29, 2021 /PRNewswire/ --The SadBaby project was recently launched to help SMA (Spinal Muscular Atrophy) patients overcome financial constraints in getting proper medical treatment.
  • The SadBaby Team aims to improve every patient's quality of life and gather sufficient funds to support the affected children and their families.
  • Working together with SMA Association in Antalya, SadBaby has already raised significant donations for babies affected with SMA and is soon organising a concert to further their funding strategy.
  • SadBaby is also planning to create an NFT marketplace where families would be able to sell NFT products and obtain financial help by this year's end.

Data for Genentech’s Evrysdi (risdiplam) Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies With Type 1 Spinal Muscular Atrophy (SMA)

Retrieved on: 
Wednesday, July 28, 2021
Research, Baby, Maternity, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Consumer, Health, Science, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, SMA, Muscle atrophy, Spinal muscular atrophies, Evrysdi® (risdiplam), SMA, Genentech in Neuroscience, Genentech, EVRYSDI® (RISDIPLAM), SMA, GENENTECH IN NEUROSCIENCE, GENENTECH

Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.

Key Points: 
  • Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.
  • We will continue to work closely with governments and the SMA community to bring Evrysdi to as many people as possible.
  • Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
  • If Evrysdi gets in ones eyes, rinse ones eyes with water
    The most common side effects of Evrysdi include:

AB Science announces the publication of new survival data showing that masitinib extended survival in ALS by 25 months relative to placebo

Retrieved on: 
Monday, July 19, 2021
Rare diseases, Motor neuron diseases, Neuromuscular Disorders, Masitinib, Amyotrophic lateral sclerosis, Branches of biology, Neuroscience

LONG-TERM FOLLOW-UP OF 75 MONTHS SHOWED THAT MASITINIB EXTENDED SURVIVAL IN AMYOTROPHIC LATERAL SCLEROSIS BY 25 MONTHS RELATIVE TO PLACEBO, PROVIDED THAT TREATMENT STARTS EARLY IN DISEASE COURSE.

Key Points: 
  • LONG-TERM FOLLOW-UP OF 75 MONTHS SHOWED THAT MASITINIB EXTENDED SURVIVAL IN AMYOTROPHIC LATERAL SCLEROSIS BY 25 MONTHS RELATIVE TO PLACEBO, PROVIDED THAT TREATMENT STARTS EARLY IN DISEASE COURSE.
  • The current confirmatory phase 3 study (AB19001) in ALS patients with mild or moderate impairment of functionality at baseline, is very well-aligned with this population.
  • This is closely aligned with the patient population that showed the greatest survival benefit with masitinib in the long-term survival analysis.
  • AB Science disclaims any obligationor undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq.

Coya Therapeutics Receives Orphan Drug Designation from the FDA for ALS001, an Autologous Treg Cell Therapy, for the Treatment of ALS

Retrieved on: 
Wednesday, July 7, 2021
Branches of biology, Rare diseases, Neuroscience, Nervous system, Motor neuron diseases, Neuromuscular Disorders, Amyotrophic lateral sclerosis, Neurological disorders, Motor neuron, Neurodegeneration, Neuron, Motor neuron disease

ALS is a progressive neurodegenerative disease primarily affecting motor neurons in the cortex, brain stem and spinal cord.

Key Points: 
  • ALS is a progressive neurodegenerative disease primarily affecting motor neurons in the cortex, brain stem and spinal cord.
  • As a result, symptoms include the loss of mobility, muscle control, speech and eventually the ability to breathe.
  • According to the ALS Association, approximately 5,000 people are diagnosed per year, and the average life expectancy is two to five years.
  • Headquartered in Houston, TX, Coya Therapeutics (TM) is a clinical-stage biotechnology company developing first-in-class and best-in-class approaches utilizing adoptive regulatory T cells (Tregs) to target disease.

MTPA Announces PANTHERx® Selected as Exclusive Provider of U.S. Specialty Pharmacy Services for EXSERVAN™ (riluzole) Oral Film

Retrieved on: 
Friday, June 25, 2021
Drugs, Neurological disorders, Branches of biology, Benzothiazoles, Riluzole, Motor neuron diseases, Neuromuscular Disorders, Mitsubishi Tanabe Pharma, Pharmacy, Amyotrophic lateral sclerosis, Specialty pharmacy

JERSEY CITY, N.J., June 25, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced PANTHERx Rare Pharmacy has been selected as the exclusive provider of specialty pharmacy services for EXSERVAN(riluzole), an oral film formulation of riluzole for the treatment of amyotrophic lateral sclerosis (ALS).

Key Points: 
  • JERSEY CITY, N.J., June 25, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced PANTHERx Rare Pharmacy has been selected as the exclusive provider of specialty pharmacy services for EXSERVAN(riluzole), an oral film formulation of riluzole for the treatment of amyotrophic lateral sclerosis (ALS).
  • Upon enrollment, PANTHERx will provide patients with a review and summary of insurance benefit options, including information about eligibility for the Out-of-Pocket Assistance Program for EXSERVAN.
  • "PANTHERx prides itself on being focused on people living with the implications of rare disease," said Rob Snyder, President of PANTHERx.
  • EXSERVAN, anoral film formulation of riluzole, was approved by the U.S. Food and Drug Administration (FDA) in November 2019.