Department of Neurology, Xuanwu hospital

Pharma Two B Announces Publication of Positive Phase 3 Study Results of P2B001, an Investigational Treatment for Early Parkinson’s Disease, in Movement Disorders

Retrieved on: 
Thursday, November 9, 2023
UPDRS, Incidence, PD, III, Neurology, University of South Florida, Physician, Emeritus, Pramipexole, Epworth Sleepiness Scale, FRCPC, LSM, ESS, Somnolence, University, Department of Neurology, Xuanwu hospital, Mount Sinai School, NDA, Safety, Patient, PPX, Movement disorder, Dietary supplement, Pharmaceutical industry, Surveying

The study included 544 untreated patients with Parkinson’s disease who were randomized (2:2:2:1) to treatment with P2B001, its individual components (pramipexole-ER 0.6 mg or rasagiline-ER 0.75 mg), or marketed PramiER individually titrated to optimal dose (1.5-4.5 mg).

Key Points: 
  • The study included 544 untreated patients with Parkinson’s disease who were randomized (2:2:2:1) to treatment with P2B001, its individual components (pramipexole-ER 0.6 mg or rasagiline-ER 0.75 mg), or marketed PramiER individually titrated to optimal dose (1.5-4.5 mg).
  • Further, he stated that “P2B001 is a once-a-day treatment that requires no titration.
  • P2B001 was generally well tolerated and showed fewer sleep-related and dopaminergic adverse events in comparison to commercially used doses of pramipexole ER.
  • “We look forward to moving P2B001 forward towards an NDA submission to make this potential treatment option available to patients.”

Nido Biosciences Presents Clinical Data Supporting the Continued Development of NIDO-361 in SBMA Patients at the 2023 International Kennedy's Disease Patient Conference

Retrieved on: 
Sunday, November 5, 2023
Science, Neurology, Radiology, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, MRI, Phase II, SBMA, University College London, International, NINDS, Treatment, Observational study, Conference, HIV disease progression rates, UCL, Pharmacokinetics, Male, Medicine, Interim, Department of Neurology, Xuanwu hospital, NIH, Spinal and bulbar muscular atrophy, DSM-IV codes, Safety, CMO, Patient, Medical imaging, Stroke, Spine

The company believes data from both studies support the advancement of the clinical evaluation of NIDO-361 in patients.

Key Points: 
  • The company believes data from both studies support the advancement of the clinical evaluation of NIDO-361 in patients.
  • NIDO-361 is in development for the treatment of patients suffering from Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s disease.
  • The novel small molecule binds to a distinct site on the androgen receptor and corrects transcriptional dysregulation to restore healthy cell function.
  • Additionally, Nido Bio has announced the completion of interim analyses in patients with SBMA enrolled in an ongoing observational study.

Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium

Retrieved on: 
Thursday, October 19, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, PSP, ORION, Progressive supranuclear palsy, Movement disorder, Pan Pacific Hotels and Resorts, Medication, MDS, Neurology, AMX0035, Disease, Aggregation, Ludwig Maximilian University of Munich, Unfolded protein response, HIV disease progression rates, LMU, Quality of life, Department of Neurology, Xuanwu hospital, Caregiver burden, Safety, Hospital, Pharmaceutical industry, Medical imaging, Amylyx Pharmaceuticals, MD

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).
  • Neuro2023: The PSP and CBD International Research Symposium will take place on October 19-20, 2023, at the Pan Pacific Hotel in London, UK.
  • The ORION Phase 3 trial was designed and planned in collaboration with key global academic leaders, people living with PSP, and industry advocacy groups.
  • Additional details regarding trial enrollment and eligibility criteria will be shared upon initiation of the trial, which is anticipated to start by the end of 2023.

Mapi Pharma to Present Plans for Additional Studies of GA Depot in Multiple Sclerosis and Other Indications and Highlight Latest Results on GA Depot in Relapsing Multiple Sclerosis at the ECTRIMS-ACTRIMS® Meeting in Milan, Italy October 11-13, 2023

Retrieved on: 
Tuesday, October 10, 2023
Congress, Mount Sinai, RMS, PPMS, NESS, Great America Committee, MS, Treatment, Multiple sclerosis, Safety, Principal, Conditional sentence, GA, Research, New Drug Application, Glatiramer acetate, Department of Neurology, Xuanwu hospital, Conference, Icahn School of Medicine at Mount Sinai, Patient, Pharmaceutical industry

Multiple sclerosis (MS) is a chronic disease requiring lifelong therapy.

Key Points: 
  • Multiple sclerosis (MS) is a chronic disease requiring lifelong therapy.
  • Glatiramer acetate (GA) long-acting injection (GA Depot) consists of extended-release GA, administered once every 28 days.
  • Ehud Marom, Chairman and Chief Executive Officer, Mapi Pharma said, “We are progressing with our New Drug Application for GA Depot for the treatment of RMS.
  • The ECTRIMS-ACTRIMS meeting is an important forum to share with the neurological and investor community that GA Depot may offer an effective treatment option to patients with relapsing forms of multiple sclerosis."

BridgeBio Pharma Shares Positive Long-Term Data from an Ongoing Phase 2 Study, which Support the Potential Use of Glycosylated Alpha-dystroglycan (⍺DG) Levels as a Surrogate Endpoint in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Retrieved on: 
Monday, October 9, 2023
R9, Cancer, WMS, Food, Biomarker, Neurology, NSAD, Safety, VCU, Month, FDA, Accelerated approval (FDA), Virginia Commonwealth University, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ClinicalTrials.gov, Creatine kinase, Department of Neurology, Xuanwu hospital, Patient, PALO, Medical imaging, Pharmaceutical industry

PALO ALTO, Calif., Oct. 09, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, shared new long-term data from its Phase 2 trial in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) at the Annual Congress of World Muscle Society (WMS) in Charleston, South Carolina. The new long-term data remains consistent with earlier data from the Phase 2 study showing a well-tolerated safety profile and encouraging preliminary efficacy. Additionally, early changes in glycosylated ⍺DG levels at 3 months appear to be associated with ambulatory improvements at 9 months, providing support for the possible use of glycosylated ⍺DG levels as a surrogate endpoint in the ongoing Phase 3 study for accelerated approval.

Key Points: 
  • The new long-term data remains consistent with earlier data from the Phase 2 study showing a well-tolerated safety profile and encouraging preliminary efficacy.
  • BridgeBio presented 21-month results from its ongoing Phase 2 trial, including:
    “There is a serious unmet need for those with LGMD2I/R9.
  • FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval.
  • The NSAD and secondary endpoints will be evaluated at 36 months, and results are expected to provide confirmatory clinical data.

NYU Langone Health Marks Historic 75th Anniversary of Rusk Rehabilitation

Retrieved on: 
Wednesday, October 11, 2023
Metropolitan Transportation Authority, Chicago Innovation Awards, Partnership, U.S. News & World Report, Physician, News, Doctor of Philosophy, Burke Rehabilitation Hospital, Rehabilitation, Amputation, United States Department of Veterans Affairs, Howard A. Rusk, Concussion, Injury, Brain, NYU Langone Health, Department of Neurology, Xuanwu hospital, MedStar National Rehabilitation Hospital, Cancer, Lasker-Bloomberg Public Service Award, Physical medicine and rehabilitation, Accessibility of the Metropolitan Transportation Authority, Howard, Te Putanga Toi Arts Access Awards, Diversity, Neurology, MPA, Edward Hines Jr. Veterans Administration Hospital, NYU, World War II, Patient, Quality of life, Hospital, Bellevue Hospital, MD, Rusk

NEW YORK, Oct. 11, 2023 /PRNewswire/ -- To commemorate 75 years of innovation in physical and rehabilitation medicine, NYU Langone's Rusk Rehabilitation held a diamond anniversary recognition on September 22, during National Rehabilitation Awareness Week, to celebrate the groundbreaking concept of whole-person care developed by Howard A. Rusk, MD, during World War II.

Key Points: 
  • Dr. Batavia, who received the Distinguished Alumni Award, is an associate professor in the physical therapy program at NYU and a former department chair.
  • "At Rusk Rehabilitation, we continue the legacy of Dr. Rusk, using the latest technology and practices to improve patient outcomes and quality of life," said Steven R. Flanagan, MD , the Howard A. Rusk Professor of Rehabilitation Medicine and chair of the Department of Rehabilitation Medicine at NYU Langone.
  • From that first center in 1948, Rusk Rehabilitation has expanded significantly over the past decades, serving 2,000 patients across 27 locations annually.
  • With 780 clinicians on staff, Rusk Rehabilitation is one of the largest facilities in the country and has trained nearly 1,000 physicians in physical medicine and rehabilitation.

Icahn School of Medicine at Mount Sinai Licenses Next-Generation Diagnostic Test Epigenetic /AI Platform to GNOMX Corp.

Retrieved on: 
Monday, September 18, 2023
Infection, Yale School of Medicine, Business development, DARPA, University, Lyme disease, Planetary Observer program, ECHO, Prognosis, Research, COVID, University of Pittsburgh School of Medicine, Department of Neurology, Xuanwu hospital, Doctor of Philosophy, Princeton University, Icahn School of Medicine at Mount Sinai, Sepsis, MSIP, Patient, Artificial intelligence, Diagnosis, Policy, Health, Mount Sinai Health System, Wilfrid Sellars, Epigenetics, Gene, Vaccine, Pharmaceutical industry, Management, MD, Neurology, Medicine

NEW YORK, Sept. 18, 2023 /PRNewswire-PRWeb/ -- The Icahn School of Medicine at Mount Sinai has agreed to an exclusive license option with GNOMX Corp. (GNOMX) for epigenetic diagnostic and prognostic technology for infectious diseases.

Key Points: 
  • The Icahn School of Medicine at Mount Sinai has agreed to an exclusive license option with GNOMX Corp. (GNOMX) for epigenetic diagnostic and prognostic technology for infectious diseases.
  • NEW YORK, Sept. 18, 2023 /PRNewswire-PRWeb/ -- The Icahn School of Medicine at Mount Sinai has agreed to an exclusive license option with GNOMX Corp. (GNOMX) for epigenetic diagnostic and prognostic technology for infectious diseases.
  • The GNOMX epigenetic diagnostic platform includes technology developed by Mount Sinai faculty and was optioned by Mount Sinai to GNOMX.
  • Mount Sinai and Mount Sinai faculty, including Dr. Sealfon, have a financial interest in this technology and in GNOMX pursuant to the Mount Sinai Intellectual Property Policy.

Teleneurology Challenges Met by Training Curriculum

Retrieved on: 
Wednesday, September 6, 2023
Seizure, Telehealth, Kurzweil, Education, Face, Stroke, Coronavirus, Curriculum, Accessory nerve disorder, NYU, New York University Grossman School of Medicine, Hypoesthesia, Neurology, Boston University, Department of Neurology, Xuanwu hospital, Pain, NYU Langone Health, Literature, Johns Hopkins University, MD, Patient, Physician, Faculty, Pharmaceutical industry, Nursing

NEW YORK, Sept. 6, 2023 /PRNewswire/ -- A new physician-training system in telehealth simulates key parts of traditional, in-person neurological exams that use little reflex hammers, pinpricks, and flashlights to test nerve function. The three-year program, which was designed by researchers at NYU Grossman School of Medicine, has trained at least 68 neurology residents since its rollout in 2020. Published online Aug. 3 in Neurology Education, a new analysis of the curriculum identifies challenges to translating in-person exam techniques that use hands-on tools to an online setting, and proposes solutions.

Key Points: 
  • Published online Aug. 3 in Neurology Education, a new analysis of the curriculum identifies challenges to translating in-person exam techniques that use hands-on tools to an online setting, and proposes solutions.
  • While virtual healthcare technology has existed in some form for decades, physicians still receive little formal training in its use.
  • According to the authors of the new report, this poses challenges for specialties like neurology that rely on physical interactions and sometimes subtle reactions by patients.
  • To develop a teleneurology curriculum that overcomes these challenges, the researchers included a range of educational methods such as reading materials, supervised clinical visits, and prerecorded role-play demonstrations performed by faculty members to showcase best practices.

Alterity Therapeutics Presents New Data on Multiple System Atrophy, a Rare Parkinsonian Disorder

Retrieved on: 
Thursday, August 31, 2023
PD, Multiple system atrophy, In vivo magnetic resonance spectroscopy, Pathology, Vanderbilt University Medical Center, Department of Neurology, Xuanwu hospital, ASX, Brain, Gliosis, Inositol, Movement disorder, Biomarker, Doctor of Philosophy, NAA, MDS, Congress, MRI, MRS, ATH, Patient, MSA, SAN, Diagnosis, Biomusic, Therapy, International Congress on Tuberculosis, MI, Disease, Medical imaging, Alterity, Neurology

MELBOURNE, Australia and SAN FRANCISCO, Aug. 31, 2023 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that presentations from its bioMUSE natural history study of Multiple System Atrophy (MSA) were delivered at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) taking place August 27 - 31, 2023 in Copenhagen, Denmark.

Key Points: 
  • The posters presented from Alterity’s bioMUSE study address the need for incorporating biomarkers as a critical component for diagnosis of MSA.
  • The diagnosis of early MSA can be challenging as individuals often present similarly to Parkinson’s disease (PD).
  • In contrast to PD, MSA is rapidly progressive and, therefore, it is vital to accurately diagnose patients enrolling in clinical trials.
  • The poster entitled, “A multimodal approach for diagnosis of early Multiple System Atrophy” was presented by Dr. Claassen.

Novel Application of Machine Learning Approaches Predicts Gait Dysfunction in Parkinson's Disease

Retrieved on: 
Sunday, August 27, 2023
Average absolute deviation, Rectifier (neural networks), Magnetic resonance imaging, DTI, Disease, MLP, Perceptron, Recursive neural network, Department of Neurology, Xuanwu hospital, Movement disorder, Movement, DWI, Parkinson's disease, Patient, HIV disease progression rates, Machine learning, AUC, Neurology, UPDRS, International Congress on Tuberculosis, Medical imaging, PD

COPENHAGEN, Denmark, Aug. 27, 2023 /PRNewswire-PRWeb/ -- A machine learning approach from diffusion tensor imaging (DTI) multi-spectral diffusion weighted imaging (DWI) showed prediction of gait dysfunction in PD according to a study released today at the International Congress of Parkinson's Disease and Movement Disorders® in Copenhagen, Denmark.

Key Points: 
  • COPENHAGEN, Denmark, Aug. 27, 2023 /PRNewswire-PRWeb/ -- A machine learning approach from diffusion tensor imaging (DTI) multi-spectral diffusion weighted imaging (DWI) showed prediction of gait dysfunction in PD according to a study released today at the International Congress of Parkinson's Disease and Movement Disorders® in Copenhagen, Denmark.
  • Several neuroimaging studies in Parkinson's disease (PD) suggest an altered interplay between cortical and subcortical brain areas at a structural and functional level each that contributes to gait dysfunction in PD.
  • This study used 7 different quantitative DTI measures from 43 PD patients along with UPDRS III, to develop a prediction model for gait dysfunction using different machine learning approaches and 60 template regions of interest (ROIs).
  • "This study suggests that a machine learning approach of DTI analysis may have potential in predicting gait dysfunction in PD patients if confirmed by larger confirmative studies.