Neurological disorders

Exicure Announces Upcoming Neuroscience Pipeline Update at Virtual R&D Day

Retrieved on: 
Thursday, July 8, 2021
Science, Other Science, Research, Pharmaceutical, Oncology, Health, Genetics, Other Health, Neurological disorders, Branches of biology, Nervous system, Rare diseases, Lipid storage disorders, Frataxin, Proteins, Batten disease, Friedreich's ataxia, CLN3, Nav1.7, Neurodegeneration, Friedreich’s Ataxia, SCN9A, CLN3 Batten Disease, Exicure, Inc., FRIEDREICH’S ATAXIA, SCN9A, CLN3 BATTEN DISEASE, EXICURE, INC.

The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.

Key Points: 
  • The event will showcase Exicures neuroscience pipeline, including its lead program for Friedreichs Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.
  • Exicure will present new and previously unreleased preclinical data and discuss progress with XCUR-FXN, which is on track for IND filing in late Q4 2021.
  • Additionally, Exicure will discuss its perspective on XCUR-FXNs competitive differentiation in FA and the programs path to clinical validation.
  • Also, Exicure will provide an update on its expanding pipeline across several rare neurodegenerative diseases of high unmet need and highlight progress with two preclinical programs targeting SCN9A (Nav1.7) for neuropathic pain and CLN3 for Batten Disease.

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.

Rare Disease Symptoms as a Marker for Rare Disease & Improving Diagnosis

Retrieved on: 
Tuesday, July 6, 2021
Branches of biology, Medicine, Neurological disorders, Rare diseases, Signs and symptoms, Rare disease, Disease, Amyotrophic lateral sclerosis, Metabolic myopathy, Neuromyotonia

Symptoms can affect any part of the body and body system, and many rare diseases present with symptoms that affect multiple parts of the body.

Key Points: 
  • Symptoms can affect any part of the body and body system, and many rare diseases present with symptoms that affect multiple parts of the body.
  • Symptoms of a rare disease can range from mild to severe, depending on the rare disease, but also on the individual affected.
  • Sometimes, even siblings or family members with the same rare disease diagnosis, display slightly different symptoms, or symptoms of varying degrees of severity.
  • Rare diseases are generally not treatable, but many of their symptoms may be, depending on the type and severity.

The Place for Children with Autism Announces Opening of Elgin Location

Retrieved on: 
Thursday, July 1, 2021
Autism, Psychiatric diagnosis, Psychiatry, Branches of biology, Neurological disorders

CHICAGO, June 30, 2021 /PRNewswire/ -- The Place for Children with Autism, a leading provider ofApplied Behavior Analysis (ABA) therapy for children on the spectrum, announces the opening of a new center in Elgin, IL on July 6, 2021.

Key Points: 
  • CHICAGO, June 30, 2021 /PRNewswire/ -- The Place for Children with Autism, a leading provider ofApplied Behavior Analysis (ABA) therapy for children on the spectrum, announces the opening of a new center in Elgin, IL on July 6, 2021.
  • The center is opening at 2450 Westfield Drive, Elgin, IL 60124 and boasts 9,500 square feet with six classrooms, an open gym, activities area, and ample outdoor space with playgrounds.
  • The Place for Children with Autismprovides a comprehensive approach to autism treatment that focuses on the highest quality therapy to enrich the lives of children with autism and their families across all communities.
  • The Place for Children with Autism works with families from all economic backgrounds to find options that fit their needs.

Azzur Group Teams with ALS TDI on Golf Tournament to Benefit ALS Therapy

Retrieved on: 
Wednesday, June 30, 2021
Neurological disorders, Neurodegenerative disorders, Amyotrophic lateral sclerosis, ALS Therapy Development Institute

VISTA, Calif., June 30, 2021 /PRNewswire/ --Azzur Cleanrooms on Demand and Azzur Group, market-leading providers for early-phaselife sciencesGxP manufacturing, is embracing its commitment to corporate social responsibility by teaming up with ALS Therapy Development Institute (ALS TDI) for the Azzur Fore ALS golf tournament to raise money for treatment of amyotrophic lateral sclerosis (ALS), aprogressive neurodegenerative disease also known as Lou Gehrig's Disease.

Key Points: 
  • VISTA, Calif., June 30, 2021 /PRNewswire/ --Azzur Cleanrooms on Demand and Azzur Group, market-leading providers for early-phaselife sciencesGxP manufacturing, is embracing its commitment to corporate social responsibility by teaming up with ALS Therapy Development Institute (ALS TDI) for the Azzur Fore ALS golf tournament to raise money for treatment of amyotrophic lateral sclerosis (ALS), aprogressive neurodegenerative disease also known as Lou Gehrig's Disease.
  • Azzur has joined forceswith CleanSpace, a national cleanrooms solutions provider, as title sponsors for the July tournament.
  • The golf tournament will be held on Thursday, July 15,2021, starting at 7:30 am PST at Goat Hill Park Golf Club in Oceanside, California.
  • The tournament proceeds will benefit the ALS TDI, the nation's only non-profit biotechnology dedicated to advances and treatments for the disease.

Discovery of the role of a key gene in the development of ALS

Retrieved on: 
Wednesday, June 30, 2021
Branches of biology, Biology, Neurological disorders, C9orf72, Amyotrophic lateral sclerosis, TAR DNA-binding protein 43, Mutation, Genetics of amyotrophic lateral sclerosis, UBQLN2

The loss of function of an important gene, C9orf72, may affect communication between motor neurons and muscles in people with this disease.

Key Points: 
  • The loss of function of an important gene, C9orf72, may affect communication between motor neurons and muscles in people with this disease.
  • A mutation in the C9orf72 gene is the primary genetic cause of ALS.
  • The study showed the effect of the loss of function induced by the mutation of the C9orf72 gene on communication between motor neurons and muscles.
  • The research group also revealed the gene's role on the protein TDP-43 (transactive response DNA binding protein 43) which plays an important role in ALS.

NeuroPace Awarded Five-Year NIH Grant Funding of More than $9M to Study RNS System in Patients with Lennox-Gastaut Syndrome

Retrieved on: 
Wednesday, June 30, 2021
Branches of biology, Neuroscience, Neurotechnology, Neuroprosthetics, Neurological disorders, Epilepsy, Responsive neurostimulation device, Neuromodulation, RNS

The study funded by the grant will explore the potential of NeuroPaces brain-responsive neuromodulation technology as a new therapy for persons living with LGS.

Key Points: 
  • The study funded by the grant will explore the potential of NeuroPaces brain-responsive neuromodulation technology as a new therapy for persons living with LGS.
  • If the RNS System meets certain criteria, experience from the study will inform the design of a future larger clinical study.
  • The RNS System, a paradigm-shifting treatment for drug-resistant focal epilepsy, is the only brain-responsive neuromodulation system approved by the FDA.
  • In 2013, it introduced the RNS System, the first and only FDA-approved closed-loop, brain-responsive neuromodulation system that delivers truly personalized, data-driven treatment.

SwanBio Therapeutics Announces First Patient Enrolled in Natural History Study to Evaluate Patients with Adrenomyeloneuropathy

Retrieved on: 
Tuesday, June 29, 2021
Mental health, Health, Genetics, Physical therapy, Other Health, General Health, Clinical trials, Neurological disorders, Health, Rare diseases, Leukodystrophies, Peroxisomal disorders, Adrenoleukodystrophy, ALD, Clinical trial, AMN, Adrenomyeloneuropathy, SwanBio Therapeutics, ADRENOMYELONEUROPATHY, SWANBIO THERAPEUTICS

CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.

Key Points: 
  • CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD) occurring in adulthood.
  • This observational, multinational study will prospectively evaluate patients to assess the course of the disease and provide insights into potential endpoints and designs for future clinical trials.
  • We are pleased to initiate this natural history study for AMN, which has been strategically designed with patients in mind, and allows for the majority of monitoring to be conducted virtually from each patients home.
  • The global study will enroll approximately 80 adult male patients with a confirmed diagnosis of adrenoleukodystrophy (ALD).

Neuros Medical Receives FDA Breakthrough Device Designation for its Novel Altius® High Frequency Nerve Block System

Retrieved on: 
Tuesday, June 29, 2021
Branches of biology, Clinical medicine, Medicine, Acute pain, Pain, Neurological disorders, Amputations, Nociception, Neuromodulation, Phantom limb, Gabapentinoid, Limb telescoping

Chronic post-amputation pain is a significant unmet medical need, as existing treatment options are limited, and consist primarily of opioids and gabapentinoids.

Key Points: 
  • Chronic post-amputation pain is a significant unmet medical need, as existing treatment options are limited, and consist primarily of opioids and gabapentinoids.
  • Neuros anticipates completion of enrollment in its pivotal QUEST (High-FreQUEncy Nerve Block for PoST-Amputation Pain) study by the Fall of 2021.
  • QUEST is designed to assess the safety & effectiveness of the Company's AltiusHigh-Frequency Nerve Block system to treat intractable chronic post-amputation pain.
  • Neuros Medical, a neuromodulation company, has developed the Altius system for the treatment of chronic post-amputation pain (Phantom Limb Pain and Residual Limb Pain).

Addex Therapeutics Initiates Pivotal Phase 2b/3 Study with Dipraglurant for Dyskinesia Associated with Parkinson’s Disease

Retrieved on: 
Tuesday, June 29, 2021
Neurological disorders, Organic compounds, Parkinson's disease, Branches of biology, Dipraglurant, Imidazopyridines, Organofluorides, Levodopa-induced dyskinesia, Catecholamines, Dyskinesia, L-DOPA

We also expect to start an exploratory Phase 2 study with dipraglurant in blepharospasm patients in the coming months.

Key Points: 
  • We also expect to start an exploratory Phase 2 study with dipraglurant in blepharospasm patients in the coming months.
  • In a Phase 2a study dipraglurant met its primary endpoint by being generally well tolerated and showing no clinically significant safety issues.
  • However, the development of dyskinesia which may be associated with long-term use of levodopa can become as disabling as the symptoms of Parkinson's disease.
  • Addexs lead product candidate, dipraglurant (mGlu5 negative allosteric modulator or NAM) is in a pivotal registration clinical trial for Parkinsons disease levodopa induced dyskinesia (PD-LID).