Neurological disorders

Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 Spinal Muscular Atrophy (SMA) at the 2021 Virtual SMA Research & Clinical Care Meeting

Retrieved on: 
Friday, June 11, 2021
Health, Other Health, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Branches of biology, Minerals, Neurological disorders, Spinal muscular atrophy, Orphan drugs, Antisense RNA, Nusinersen, Myostatin, SMA, Topaz, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

SMA remains a devastating and debilitating disease despite the utilization of SMN upregulators that prevent further motor neuron deterioration.

Key Points: 
  • SMA remains a devastating and debilitating disease despite the utilization of SMN upregulators that prevent further motor neuron deterioration.
  • Scholar Rocks TOPAZ Phase 2 trial (NCT03921528) evaluated apitegromab across a broad age range (2-21 years) of patients with Type 2 and 3 SMA.
  • With the exception of some ambulatory patients who received apitegromab as a monotherapy, patients enrolled in TOPAZ were receiving chronic maintenance doses of nusinersen.
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.

Acorn Health Acquires LEAP Behavior Analysis in Tennessee

Retrieved on: 
Thursday, June 10, 2021
Children, Health, Mental health, Consumer, Mind, Psychology, Self, Psychiatric diagnosis, Autism, Learning disabilities, Neurological disorders, Behaviorism, Autism spectrum, Applied behavior analysis, Acorn Computers, Draft:Hopebridge Autism Therapy Centers

Acorn Health, a national provider of Applied Behavior Analysis (ABA) therapy for children diagnosed with Autism Spectrum Disorder (ASD), is pleased to announce the acquisition of substantially all of the assets of LEAP Behavior Analysis (LEAP or the Company).

Key Points: 
  • Acorn Health, a national provider of Applied Behavior Analysis (ABA) therapy for children diagnosed with Autism Spectrum Disorder (ASD), is pleased to announce the acquisition of substantially all of the assets of LEAP Behavior Analysis (LEAP or the Company).
  • The Company is now a wholly-owned subsidiary of Acorn Health of Tennessee, and will operate under the Acorn Health brand.
  • We are excited to welcome LEAP into the Acorn Health network of high-quality ABA centers, said Vicki Kroviak, CEO of Acorn Health.
  • Acorn Health is a national provider of applied behavior analysis (ABA) therapy for children diagnosed with Autism Spectrum Disorder with locations in Michigan, Illinois, Virginia, Florida, Maryland, Pennsylvania, and Tennessee.

Gain Therapeutics Announces Issuance of Two New PCT Patents Covering GBA and GALC Modulator Compounds for Treatment of CNS Disorders

Retrieved on: 
Thursday, June 10, 2021
Branches of biology, Neurological disorders, Biology, Cognitive disorders, Dementia, Amyloidosis, Lipid storage disorders, Lewy body dementias, Proteopathy, Gaucher's disease, Lewy, Glucocerebrosidase

PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinsons Disease, Lewy body dementia and Alzheimers.

Key Points: 
  • PCT/IB2020/061158 and PCT/IB2020/0611156 are directed at compounds targeting misfolded beta-glucocerebrosidase (GBA) and galactosylceramidase (GALC) respectively, addressing central nervous system (CNS) and demyelinating disorders such as Parkinsons Disease, Lewy body dementia and Alzheimers.
  • PCT/IB2020/061158 covers compounds and therapeutic uses of conditions associated with the alteration of Beta-glucocerebrosidase (GBA).
  • These conditions include Gaucher Disease, Parkinsons Disease, and other Lewy body disorders including Alzheimers Disease and amyotrophic lateral sclerosis (ALS).
  • By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding.

Trevi Therapeutics to Present at 2021 BIO Digital Conference

Retrieved on: 
Wednesday, June 9, 2021
Parkinson's disease, Branches of biology, Chemical compounds, Organ systems, Analgesics, Neurological disorders, Opioid antagonists, Tertiary alcohols, Dyskinesia, Levodopa-induced dyskinesia, Opioid, Nalbuphine

NEW HAVEN, Conn., June 9, 2021 /PRNewswire/ -- Trevi Therapeutics, Inc. (Nasdaq: TRVI), a clinical-stage biopharmaceutical company focused on the development and commercialization of the investigational therapy Haduvio (nalbuphine ER) to treat serious neurologically mediated conditions, today announced that Jennifer Good, President and CEO, has been selected to deliver a company presentation at 2021 BIO Digital .For 2021, the BIO International Convention will be held virtually as BIO Digital, on June 10 and 11 and from June 14-18.

Key Points: 
  • NEW HAVEN, Conn., June 9, 2021 /PRNewswire/ -- Trevi Therapeutics, Inc. (Nasdaq: TRVI), a clinical-stage biopharmaceutical company focused on the development and commercialization of the investigational therapy Haduvio (nalbuphine ER) to treat serious neurologically mediated conditions, today announced that Jennifer Good, President and CEO, has been selected to deliver a company presentation at 2021 BIO Digital .For 2021, the BIO International Convention will be held virtually as BIO Digital, on June 10 and 11 and from June 14-18.
  • The Company's presentation will be available to registered BIO Digital attendees from 9am ET June 10, until the end of the conference on June 18.
  • Trevi is also developing Haduvio for the treatment of levodopa-induced dyskinesia (LID) in patients with Parkinson's disease.
  • These conditions share a common pathophysiology that is mediated through opioid receptors in the central and peripheral nervous systems.

Motor neurons derived from patients point to new possible drug target for ALS

Retrieved on: 
Tuesday, June 8, 2021
Branches of biology, Biology, Neurological disorders, Induced stem cells, Motor neuron diseases, Stem cells, Metalloproteins, Oxidoreductases, SOD1, A4V, Induced pluripotent stem cell, Amyotrophic lateral sclerosis

Researchers at Boston Children's Hospital, working in collaboration with Pfizer, now report a high-throughput target and drug discovery platform using motor neurons made from ALS patients.

Key Points: 
  • Researchers at Boston Children's Hospital, working in collaboration with Pfizer, now report a high-throughput target and drug discovery platform using motor neurons made from ALS patients.
  • To create the motor neurons used for drug screening, the team used induced pluripotent stem cells, made by the lab of Kevin Eggan at Harvard University from tissue samples of patients with ALS who carried the SOD1(A4V) mutation.
  • Woolf and colleagues previously showed that human motor neurons with ALS mutations are more excitable than normal motor neurons.
  • After three rounds of screening, they found 67 compounds that reduced the hyperexcitability of the patient-derived motor neurons, without causing toxicity.

Cell Reports Publishes Discovery Data Supporting the Importance of Ion Channel, Kv7.2/7.3 as a Therapeutic Target in ALS

Retrieved on: 
Tuesday, June 8, 2021
Research, Genetics, Clinical trials, Other Health, Biotechnology, Managed care, Pharmaceutical, Health, Science, Other Science, Neurological disorders, Branches of biology, Neurodegenerative disorders, Efferent neurons, Amyotrophic lateral sclerosis, Motor neuron, Neuron, Kv7.2/7.3 Ion Channel Openers, QRL-101 (QRA-244), QurAlis Corporation, KV7.2/7.3 ION CHANNEL OPENERS, QRL-101 (QRA-244), QURALIS CORPORATION

The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.

Key Points: 
  • The publication describes a live-cell screening strategy targeting abnormal electrophysiological properties to reveal targets that modulate the intrinsic hyperexcitability of ALS motor neurons.
  • This unbiased screen using human ALS motor neurons identified Kv7.2/7.3 as a strongly overrepresented drug target.
  • Previous research has identified Kv7.2/7.3 as an ALS drug target and the unbiased nature of this screen further emphasizes the importance of Kv7.2/7.3 in ALS motor neuron dysfunction.
  • We are advancing three antisense and small molecule programs addressing sub-forms of ALS that account for the majority of ALS patients.

Aeterna Zentaris Engages Dr. Michael Levy to Support Development of Autoimmune and Inflammatory CNS Disorders Programs

Retrieved on: 
Monday, June 7, 2021
Neurological disorders, Autoimmune diseases, Central nervous system, Nervous system, Rare diseases, Neuromyelitis optica spectrum disorder, Central nervous system disorders, Myelitis

Dr. Levy will provide scientific support and advice to Aeterna Zentaris in the field of inflammatory CNS disorders, autoimmune diseases of the nervous system, and NMOSD.

Key Points: 
  • Dr. Levy will provide scientific support and advice to Aeterna Zentaris in the field of inflammatory CNS disorders, autoimmune diseases of the nervous system, and NMOSD.
  • We are pleased to welcome Dr. Levy and to have his support as we advance the development of this important program forward, commented Dr. Klaus Paulini , Chief Executive Officer of Aeterna.
  • I am excited to support Aeterna Zentaris development of AIM Biologicals and to potentially offer a new treatment for NMOSD patients, where there remains significant unmet medical need.
  • Dr. Levy is also an Associate Professor at Harvard Medical School and Director of the Neuromyelitis Optica Clinic and Research Laboratory at MGH.

Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting

Retrieved on: 
Monday, June 7, 2021
FDA, Health, Genetics, General Health, Pharmaceutical, Biotechnology, Neurological disorders, Branches of biology, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, Myostatin, SMA, Spinal muscular atrophies, SMA4, Apitegromab, SMA, Scholar Rock, APITEGROMAB, SMA, SCHOLAR ROCK

A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.

Key Points: 
  • A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.
  • Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA).
  • Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA.
  • Spinal muscular atrophy (SMA) is a rare, and often fatal, genetic disorder that typically manifests in young children.

Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2021 Peripheral Nerve Society Annual Meeting

Retrieved on: 
Friday, June 4, 2021
Amyloidosis, Branches of biology, Proteins, Anatomy, Neurological disorders, Protein folding, Histopathology, Structural proteins, Familial amyloid polyneuropathy, Transthyretin, NTLA, Amyloid

The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).

Key Points: 
  • The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).
  • Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader population of people with ATTR amyloidosis, including those with cardiomyopathy.
  • Intellia will host a live webcast on Monday, June 28, 2021 at 8:00 a.m. E.T.
  • Intellias global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

Molecules in Saliva May Hold the Key to Tracking Improvements in Autism

Retrieved on: 
Thursday, June 3, 2021
Autism, Branches of biology, Psychiatry, Abnormal psychology, Psychiatric diagnosis, Pervasive developmental disorders, Learning disabilities, Neurological disorders, Autism spectrum

SYRACUSE, N.Y., June 3, 2021 /PRNewswire/ --Molecules in the saliva may hold promise as a means to track the effectiveness of therapeutic interventions in children with autism spectrum disorder (ASD).

Key Points: 
  • SYRACUSE, N.Y., June 3, 2021 /PRNewswire/ --Molecules in the saliva may hold promise as a means to track the effectiveness of therapeutic interventions in children with autism spectrum disorder (ASD).
  • The results of this research was published in a paper titled "Longitudinal Stability of Salivary MicroRNA Biomarkers in Children and Adolescents with Autism Spectrum Disorder."
  • The prevalence of autism spectrum disorder has been consistently on the rise over the past few decades.
  • This novel approach in tracking RNA changes longitudinally in response to treatment can be another tool at the disposal of clinicians."