Neurological disorders

PTC Announces Regulatory Update on Risdiplam for Spinal Muscular Atrophy (SMA)

Retrieved on: 
Tuesday, April 7, 2020
Neurological disorders, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, RTT, Risdiplam, Neuromuscular disease, SMA, Olesoxime, Spinal muscular atrophies

Roche recently submitted additional data including comprehensive data from SUNFISH part 2 to help provide access to risdiplam for a broad range of people living with spinal muscular atrophy (SMA), triggering this extension.

Key Points: 
  • Roche recently submitted additional data including comprehensive data from SUNFISH part 2 to help provide access to risdiplam for a broad range of people living with spinal muscular atrophy (SMA), triggering this extension.
  • Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications.
  • As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products.
  • There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including risdiplam.

Genentech Provides Regulatory Update on Risdiplam for the Treatment of Spinal Muscular Atrophy (SMA)

Retrieved on: 
Tuesday, April 7, 2020
Biotechnology, FDA, Health, Pharmaceutical, Clinical trials, Neurological disorders, Organ systems, Neurodegenerative disorders, Motor neuron diseases, Neurogenetic disorders, Neuromuscular Disorders, RTT, Neuromuscular disease, Risdiplam, Priority review, Atrophy, SMA, SMA, risdiplam, Genentech in neuroscience, Genentech

These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy.

Key Points: 
  • These data were recently presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy.
  • In November 2019, the FDA granted Priority Review for risdiplam with a decision for approval expected by May 24, 2020.
  • Regulatory filings for approval of risdiplam have been submitted to health authorities in six other countries around the world.
  • Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications.

Axsome Therapeutics Announces AXS-07 Achieves Both Co-Primary Endpoints and Prevents Migraine Pain Progression in the INTERCEPT Phase 3 Trial in the Early Treatment of Migraine

Retrieved on: 
Monday, April 6, 2020
Migraine, Neurological disorders, Pain, Migraine treatment, Almotriptan

In the trial, AXS-07 met the co-primary endpoints of freedom from migraine pain and freedom from most bothersome symptoms as compared to placebo.

Key Points: 
  • In the trial, AXS-07 met the co-primary endpoints of freedom from migraine pain and freedom from most bothersome symptoms as compared to placebo.
  • A single dose of AXS-07 significantly prevented progression of migraine pain beyond mild intensity while significantly reducing the use of rescue medication.
  • The INTERCEPT study demonstrated high rates of freedom from migraine pain with AXS-07 treatment, and utilized an innovative design to evaluate migraine pain progression.
  • INTERCEPT (Initiating Early Control of Migraine Pain and Associated Symptoms) is a Phase 3, randomized, double-blind, multicenter, placebo-controlled trial evaluating the early treatment of migraine with AXS-07.

Muscular Dystrophy Association Announces Formation of Strategic Medical Advisory Team of Experts in Neuromuscular Care and Research

Retrieved on: 
Thursday, April 2, 2020
Neurological disorders, Organ systems, Nervous system, Amyotrophic lateral sclerosis, RTT, Muscular Dystrophy Association, Neuromuscular disease, Muscular dystrophy, Dystrophy, Neuromuscular Disorders, Lewis Rowland, ALS Society of Canada

NEW YORK, April 2, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the formation of its formal Medical Advisory Team who provide MDA with strategic guidance on issues that impact research and clinical care for people living with muscular dystrophy, amyotrophic lateral sclerosis (ALS) and related neuromuscular disorders more than 43 diseases.

Key Points: 
  • NEW YORK, April 2, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the formation of its formal Medical Advisory Team who provide MDA with strategic guidance on issues that impact research and clinical care for people living with muscular dystrophy, amyotrophic lateral sclerosis (ALS) and related neuromuscular disorders more than 43 diseases.
  • Each of the team members is a leading clinician at an MDA Care Center and is an internationally known expert in muscular dystrophy, ALS and related neuromuscular diseases.
  • He sees patients in the Eleanor and Lou Gehrig ALS Center, the Adult Muscular Dystrophy Association Clinic, the Pediatric Muscular Dystrophy Association Clinic, and until recently, the ALS Clinic of the Bronx VA Hospital.
  • MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related neuromuscular diseases.

Autism Rate Continues To Rise: One in 54 Children in the U.S. Has Autism

Retrieved on: 
Thursday, April 2, 2020
Psychiatry, Autism, Abnormal psychology, Psychiatric diagnosis, Pervasive developmental disorders, Medicine, Neurological disorders, RTT, Autism spectrum, Epidemiology of autism

Important findings in the recent report include the fact that more children are being evaluated for ASD, and at younger ages.

Key Points: 
  • Important findings in the recent report include the fact that more children are being evaluated for ASD, and at younger ages.
  • This suggests that we are doing a better job across the country identifying children in some historically under-reached communities.
  • Hispanic children also continue to be identified as having ASD less frequently than white or black children."
  • The National Autism Center at May Institute continues to offer free, downloadable resources about autism treatments at www.nationalautismcenter.org , including information about early warning signs.

Axial Biotherapeutics Announces Positive Topline Results from Phase 1b/2a Clinical Trial of AB-2004 for the Treatment of Autism Spectrum Disorder

Retrieved on: 
Thursday, April 2, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Psychiatry, Psychiatric diagnosis, Abnormal psychology, Pervasive developmental disorders, Organ systems, Neurological disorders, RTT, Autism, Spectrum disorder, Autism therapies

These initial data support the continued advancement of this program, further de-risking our future development plans.

Key Points: 
  • These initial data support the continued advancement of this program, further de-risking our future development plans.
  • I am encouraged by these early clinical findings and look forward to working with Axial on the design and execution of their Phase 2 placebo-controlled study.
  • AB-2004 is a first-in-class, orally administered therapeutic, targeting Autism Spectrum Disorder (ASD).
  • According to the Center for Disease Control (CDC), about 1 in 59 children has been identified with Autism Spectrum Disorder (ASD).

Muscular Dystrophy Association to Host MDA Frontline COVID-19 Response with Dr. Barry Byrne, a Facebook Live Q&A on Protecting Neuromuscular Disease Community in Midst of COVID-19 Pandemic

Retrieved on: 
Tuesday, March 31, 2020
Neurological disorders, Organ systems, Amyotrophic lateral sclerosis, Muscular Dystrophy Association, Neuromuscular disease, Muscular dystrophy, Dystrophy, Health, Duchenne, Muscular Dystrophy Community Assistance Research and Education Amendments, Muscular Dystrophy Canada

The neuromuscular disease community people with ALS, muscular dystrophy, SMA, Duchenne and related rare diseases are among the highest risk populations for COVID-19.

Key Points: 
  • The neuromuscular disease community people with ALS, muscular dystrophy, SMA, Duchenne and related rare diseases are among the highest risk populations for COVID-19.
  • MDA will request questions from the community in forthcoming posts on the @MDAorg Facebook page.
  • ** panelists available for interview prior to, or following, Q&A**
    Dr. Barry J. Byrne, M.D., Ph.D: Dr. Byrne is a leading voice in neuromuscular disease research and care.
  • Since 1950, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people affected by muscular dystrophy, ALS and related neuromuscular diseases.

AB Science announces that FDA clears masitinib IND in Amyotrophic Lateral Sclerosis (ALS), allowing U.S. patient enrollment to commence in Phase 3 study

Retrieved on: 
Tuesday, March 31, 2020
Drugs, Neurological disorders, Health, Masitinib, Riluzole, Investigational New Drug, Amyotrophic lateral sclerosis

FDA clears masitinib IND in Amyotrophic Lateral Sclerosis (ALS), allowing U.S. patient enrollment to commence in Phase 3 study

Key Points: 
  • FDA clears masitinib IND in Amyotrophic Lateral Sclerosis (ALS), allowing U.S. patient enrollment to commence in Phase 3 study
    AB Science SA (NYSE Euronext - FR0010557264 - AB) today announces that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application, allowing the Company to initiate its masitinib Phase 3 study (AB19001) in amyotrophic lateral sclerosis (ALS).
  • Masitinib as an Add-on Therapy to Riluzole in Patients with Amyotrophic Lateral Sclerosis: A Randomised Clinical Trial.
  • Schwann cells orchestrate peripheral nerve inflammation through the expression of CSF1, IL34, and SCF in amyotrophic lateral sclerosis.
  • Post-paralysis tyrosine kinase inhibition with masitinib abrogates neuroinflammation and slows disease progression in inherited amyotrophic lateral sclerosis.

Ovid Therapeutics Announces Initial Data with Soticlestat in CDKL5 Deficiency Disorder and Dup15q Syndrome

Retrieved on: 
Monday, March 30, 2020
Neurological disorders, Health, Organ systems, Epilepsy, Lennox–Gastaut syndrome, Dravet syndrome, Syndromes, Anticonvulsants, Chloroarenes, Epilepsy-intellectual disability in females, Epilepsy syndromes

We will work closely to evaluate the full data from the ARCADE study, expected in the first quarter of 2021.

Key Points: 
  • We will work closely to evaluate the full data from the ARCADE study, expected in the first quarter of 2021.
  • More data is needed to assess efficacy, but initial data support the potential of soticlestat to provide a clinical benefit for patientswith these ultra-rare and treatment-refractory epilepsy disorders.
  • Data from the first 11 patients suggests that soticlestat may reduce seizure frequency compared to baseline levels in individual patients.
  • The FDA has granted orphan drug designation to soticlestat for the treatment of both Dravet syndrome and Lennox-Gastaut syndrome.

electroCore Announces 510(k) Clearance of gammaCore™ Label Expansion into Migraine Prevention

Retrieved on: 
Monday, March 30, 2020
Headaches, Clinical medicine, Medicine, Migraine, Neurological disorders, Pain management, Cluster headache, Headache, Prevention of migraines, Methysergide

The American Migraine Foundation estimates that there are nearly 36 million adults in the United States suffering from migraine.

Key Points: 
  • The American Migraine Foundation estimates that there are nearly 36 million adults in the United States suffering from migraine.
  • gammaCore has been available for the acute treatment of migraine, and now can be used for prevention to more effectively treat these patients.
  • This milestone is clearly a very meaningful expansion of our label indication within our total addressable market.
  • In addition to migraine prevention, gammaCore is currently indicated for adjunctive use for the preventive treatment of cluster headache and for the acute treatment of pain associated with episodic cluster headache and migraine headache in adult patients.