Neurological disorders

Neurocrine Biosciences Announces New Data Published from the Largest Real-World Screening Study, RE-KINECT, Demonstrating that Movements Consistent with Tardive Dyskinesia Occur Frequently and Can Reduce Quality of Life in Patients with Psychiatric Disord

Retrieved on: 
Thursday, May 7, 2020
Organ systems, Neurological disorders, Nervous system, Health, Tardive dyskinesia, Movement disorders, Neurocrine Biosciences, Psychiatry, Sulpiride

"Many patients reported that involuntary movements from possible tardive dyskinesia impact their ability to talk, socialize and be productive, leaving many of them feeling self-conscious or embarrassed.

Key Points: 
  • "Many patients reported that involuntary movements from possible tardive dyskinesia impact their ability to talk, socialize and be productive, leaving many of them feeling self-conscious or embarrassed.
  • These data support the continued need to raise awareness of involuntary movements from possible tardive dyskinesia and the importance of properly screening, diagnosing and helping to relieve the suffering that many of these patients are experiencing."
  • RE-KINECT, a prospective real-world screening study that included 739 patients from 37 outpatient psychiatry practices in the U.S., was conducted with support from Neurocrine Biosciences.
  • Patients were clinically evaluated for abnormal involuntary movements in general body regions (head/face, neck/trunk, upper/lower limbs) as well as for possible TD.

Dr. Reddy's Laboratories Received Approval for Their NDA, ELYXYB™ (celecoxib oral solution) 25 mg/mL, in the U.S.

Retrieved on: 
Wednesday, May 6, 2020
FDA, Pharmaceutical, Health, Migraine, Neurological disorders, Medicine, Clinical medicine, Celecoxib, Aura, Almotriptan, Prevention of migraines

ELYXYB (previously known as DFN-15) is indicated for the acute treatment of migraine with or without aura in adults.

Key Points: 
  • ELYXYB (previously known as DFN-15) is indicated for the acute treatment of migraine with or without aura in adults.
  • Erez Israeli, CEO of Dr. Reddys Laboratories, commented, We are excited about the approval of ELYXYB.
  • In pivotal studies, ELYXYB demonstrated a rapid onset of action which is critically important to patients suffering from acute migraine attacks.
  • The results from pivotal studies with ELYXYB established efficacy of celecoxib in the treatment of migraine with very few adverse events.

Zogenix to Participate in the BofA Securities 2020 Virtual Healthcare Conference

Retrieved on: 
Wednesday, May 6, 2020
Neurological disorders, Health, Organ systems, Lennox–Gastaut syndrome, Syndromes, Epilepsy, Fenfluramine, Epilepsy-intellectual disability in females, Epilepsy syndromes

Zogenix is a global pharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.

Key Points: 
  • Zogenix is a global pharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases.
  • The company has two late-stage development programs underway: FINTEPLA (ZX008, fenfluramine oral solution) for the treatment of seizures associated with Dravet and Lennox-Gastaut syndromes, two rare and often-catastrophic childhood-onset epilepsies, and MT1621, a novel substrate enhancement therapy for the treatment of a rare genetic disorder called TK2 deficiency.

Amylyx Pharmaceuticals Gets #INKEDforALS

Retrieved on: 
Monday, May 4, 2020
Biotechnology, General Health, Mental health, Health, Pharmaceutical, Neurological disorders, Neurodegenerative disorders, Nervous system, ALS Awareness Month, Amyotrophic lateral sclerosis, ALS Society of Canada, Les Turner ALS Foundation

Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today is honored to support ALS Awareness Month 2020 with the launch of #INKEDforALS , a campaign that aims to raise awareness for ALS.

Key Points: 
  • Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today is honored to support ALS Awareness Month 2020 with the launch of #INKEDforALS , a campaign that aims to raise awareness for ALS.
  • We are honored to support ALS Awareness Month this year through the #INKEDforALS campaign, said Justin Klee, Co-CEO and Co-Founder of Amylyx.
  • With social distancing in place, this years ALS Awareness Month is especially important to us, said Joshua Cohen, Co-CEO, Chairman, and Co-Founder of Amylyx.
  • Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimers disease and other neurodegenerative diseases.

Seelos Therapeutics Receives Notice of Allowance for an Additional US Patent for Trehalose (SLS-005)

Retrieved on: 
Monday, May 4, 2020
Branches of biology, Biology, TFEB, Programmed cell death, Autophagy, Neurological disorders, Trehalose, MTOR, Lysosome, Protein aggregation

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly, activates autophagy, which is the process that clears material from cells.

Key Points: 
  • Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly, activates autophagy, which is the process that clears material from cells.
  • In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material.
  • Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression.
  • Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

Newron Reports Top-Line Results From Its STARS Study Evaluating Sarizotan in Patients With Rett Syndrome

Retrieved on: 
Monday, May 4, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Health, Neurological disorders, Organ systems, Clinical trials, Medical statistics, RTT, Psychiatric diagnosis, Pervasive developmental disorders, Clinical endpoint, Rett, Rett Syndrome, Newron Pharmaceuticals

The STARS (Sarizotan for the Treatment of Apneas in Rett Syndrome) clinical study qualified and enrolled 129 Rett syndrome patients in 14 centres throughout the US, Europe, Asia and Australia for the six-month clinical trial.

Key Points: 
  • The STARS (Sarizotan for the Treatment of Apneas in Rett Syndrome) clinical study qualified and enrolled 129 Rett syndrome patients in 14 centres throughout the US, Europe, Asia and Australia for the six-month clinical trial.
  • The primary endpoint of the STARS study was a percentage reduction in episodes of apnea during waking time compared with placebo.
  • Ravi Anand, M.D., Chief Medical Officer of Newron, commented: We are very disappointed that the top-line results in the STARS study did not meet the study endpoints.
  • We are currently awaiting results of additional explanatory analyses and will continue to analyse the full data set from the study to understand more about the results.

Marinus Pharmaceuticals Announces Formation of Scientific Advisory Board

Retrieved on: 
Thursday, April 30, 2020
Neurological disorders, Health, Epilepsy, Medicine, Ganaxolone, Epileptic seizure, Anticonvulsant, Epilepsy-intellectual disability in females

We believe the strength and enthusiasm of our newly-formed scientific advisory board, with an expertise in clinical development and global registration strategies for novel anti-epileptic drugs, is a testament to our commitment to ganaxolone in patients with severe seizure disorders, said Scott Braunstein, M.D., Chief Executive Officer of Marinus.

Key Points: 
  • We believe the strength and enthusiasm of our newly-formed scientific advisory board, with an expertise in clinical development and global registration strategies for novel anti-epileptic drugs, is a testament to our commitment to ganaxolone in patients with severe seizure disorders, said Scott Braunstein, M.D., Chief Executive Officer of Marinus.
  • The insights from these scientific thought-leaders will be invaluable as we approach key milestones in our epilepsy programs.
  • He has served on the board of directors of the American Epilepsy Society and has been a member of advisory panels to the National Institutes of Health.
  • Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders.

ProMIS Neurosciences creates novel intrabodies for ALS, frontotemporal dementia and other neurodegenerative diseases

Retrieved on: 
Thursday, April 30, 2020
Branches of biology, Proteins, Neurological disorders, Organ systems, TARDBP, Neurodegeneration, Protein aggregation, Protein folding

Intrabodies are antibody therapies intended to work inside a cell, blocking toxic proteins, accelerating their degradation and preventing their spread to healthy cells.

Key Points: 
  • Intrabodies are antibody therapies intended to work inside a cell, blocking toxic proteins, accelerating their degradation and preventing their spread to healthy cells.
  • ProMIS has generated several highly selective intrabodies that bind to toxic TDP-43 protein aggregates within cells and promote their degradation without affecting normal TDP-43.
  • Weve used our platform to produce novel antibody candidates for several neurodegenerative diseases, including Parkinsons and Alzheimers.
  • In neurodegenerative diseases, such as Alzheimers, ALS and Parkinsons disease, the DSEs are misfolded regions on otherwise normal proteins.

Prothena to Report First Quarter 2020 Financial Results on May 6

Retrieved on: 
Wednesday, April 29, 2020
Branches of biology, Organ systems, Biology, Neurological disorders, Protein folding, RTT, Histopathology, Proteopathy, Transthyretin, Amyloid beta, Amyloidosis, Alzheimer's disease

DUBLIN, Ireland, April 29, 2020 (GLOBE NEWSWIRE) -- Prothena Corporation plc (NASDAQ:PRTA), a clinical-stage neuroscience company with expertise in protein misfolding, announced today that it will report its first quarter of 2020 financial results on Wednesday, May 6, 2020, after the close of the U.S. financial markets.

Key Points: 
  • DUBLIN, Ireland, April 29, 2020 (GLOBE NEWSWIRE) -- Prothena Corporation plc (NASDAQ:PRTA), a clinical-stage neuroscience company with expertise in protein misfolding, announced today that it will report its first quarter of 2020 financial results on Wednesday, May 6, 2020, after the close of the U.S. financial markets.
  • Consistent with past practice, the Company will not be conducting a conference call in conjunction with this financial results release on May 6.
  • Prothenas proprietary programs include PRX004 for the potential treatment of ATTR amyloidosis, and programs that target A (Amyloid beta) for the potential treatment of Alzheimers disease.
  • For more information, please visit the Companys website at www.prothena.com and follow the Company on Twitter @ProthenaCorp.

Muscular Dystrophy Association's COVID-19 Emergency Fund Launches Extensive Campaign in ALS Awareness Month to Raise Funds for the Most Vulnerable During the Pandemic

Retrieved on: 
Wednesday, April 29, 2020
Neurological disorders, Organ systems, Amyotrophic lateral sclerosis, Muscular Dystrophy Association, MDA, Dystrophy, Neuromuscular disease, Health, Muscular dystrophy

This webinar is led by Dr. Ambereen Mehta and Hillary Zebberman, MSW, LCSW, from UCLA Medical Center and is available here .

Key Points: 
  • This webinar is led by Dr. Ambereen Mehta and Hillary Zebberman, MSW, LCSW, from UCLA Medical Center and is available here .
  • The live Q&A event, open to a global ALS audience, is slated for May 1 at 3 p.m.
  • To continue to raise critical emergency funds, MDA is introducing a weekend filled with nonstop virtual programming in support of the organization's mission.
  • For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases.