Narcolepsy

Harmony Biosciences Announces Funding for Unique Community Initiatives Through its Patients and Progress at the Heart Awards Programs

Retrieved on: 
Tuesday, July 25, 2023
Society, Mariposa, Heart, DSM-IV codes, Progress, Doctor of Philosophy, The Progress, Mother, Development, Patient, Society for Women's Health Research, Lactation, Empathy, Neurology, Introduction, Corporate Affairs, Pregnancy, University, Impact, Idiopathic hypersomnia, Community, IH, Research, Organization, PAP, Knowledge, Diagnosis, FDA, Sleep, Conference, Social support, Overalls, Public, Harmony, Stigma, Sleep disorder, Letter, Caregiver, Caregiver stress, Drug development, Focus group, Health, Pharmaceutical industry, Birth control, Nursing, Medical imaging, Medical device, Narcolepsy, Woman

Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases.

Key Points: 
  • Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases.
  • "With the support of this funding, we can advance our educational program 'The Impact of Narcolepsy on Women's Health Across the Lifespan.'
  • Progress at the Heart provides funding support for novel initiatives addressing disparities, injustices, and inequities in the rare neurological disease and sleep disorder communities.
  • Harmony will announce recipients for the next round of Patients and Progress at the Heart awards in October 2023, continuing its commitment to the rare neurological and sleep disorder communities.

HARMONY BIOSCIENCES ANNOUNCES PLANS TO INITIATE PHASE 3 REGISTRATIONAL STUDY OF PITOLISANT IN PRADER-WILLI SYNDROME FOLLOWING POSITIVE END-OF-PHASE 2 MEETING WITH THE U.S. FOOD AND DRUG ADMINISTRATION

Retrieved on: 
Thursday, July 20, 2023
Prader–Willi syndrome, Cataplexy, Narcolepsy, EDS, Pitolisant, DSM-IV codes, Harmony, MD, Patient, Somnolence, PWS, FDA, Safety, Pharmaceutical industry

Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023.

Key Points: 
  • Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023.
  • Based on a positive signal observed from the Phase 2 proof-of-concept study, Harmony aligned with the FDA on the proposed Phase 3 study design elements to support further investigation of pitolisant for children, adolescents and adults with PWS experiencing EDS.
  • Pitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy.
  • Pitolisant is not approved for use in patients with PWS and is currently being evaluated as an investigational agent in this patient population.

Wake Up Narcolepsy Debuts Public Service Announcement in Collaboration with Red Square Pictures

Retrieved on: 
Tuesday, July 11, 2023
Interim, Diagnosis, Partnership, Research, PSA, Wake Up, WUN, Disease, Pharmaceutical industry, Narcolepsy

WORCESTER, Mass., July 11, 2023 (GLOBE NEWSWIRE) -- Wake Up Narcolepsy (WUN), a national leader for research and awareness, premiered its Narcolepsy public service announcement in partnership with Red Square Pictures.

Key Points: 
  • WORCESTER, Mass., July 11, 2023 (GLOBE NEWSWIRE) -- Wake Up Narcolepsy (WUN), a national leader for research and awareness, premiered its Narcolepsy public service announcement in partnership with Red Square Pictures.
  • The informational PSA aims to accurately depict Narcolepsy and how debilitating this rare medical disorder can be.
  • “It’s our hope that this PSA will help bring about a greater public understanding of Narcolepsy while also offering support to those facing this challenging diagnosis.”
    Wake Up Narcolepsy announced a media partnership with Red Square Pictures last fall to benefit people living with Narcolepsy.
  • To that end, Red Square Pictures is currently developing a full-length documentary feature film.

Beacon Biosignals Acquires Dreem, Launches At-home Sleep Monitoring Services for Clinical Trials

Retrieved on: 
Tuesday, July 11, 2023
DSM-IV codes, Actigraphy, Drug development, Sleep, Hypersomnia, Arnal, Schizophrenia, Clinical trial, Insomnia, Quality of life, Narcolepsy, Research, Brain, Biomarker, Disorder, Science, Neuropsychiatric Disease and Treatment, Acquisition, MD, Machine learning, EEG, Beacon, Alzheimer's disease, Medical device, Pharmaceutical industry, Medical imaging, Bad Dreems

Dreem, a market leader in developing clinically-validated sleep monitoring devices, is poised to markedly enhance Beacon's capabilities in data acquisition and clinical trial operations, creating substantial value for Beacon's biopharma partners.

Key Points: 
  • Dreem, a market leader in developing clinically-validated sleep monitoring devices, is poised to markedly enhance Beacon's capabilities in data acquisition and clinical trial operations, creating substantial value for Beacon's biopharma partners.
  • As part of this acquisition, Beacon Biosignals secured an undisclosed financing round with participation from its syndicate of Series A investors, led by General Catalyst.
  • At-home, longitudinal monitoring is positioned to progress the underlying science of sleep physiology beyond what is possible with limited assessments in an unnatural sleep lab setting.
  • Prominent sleep expert and Beacon Biosignals Scientific Advisory Board member, Dr. Andrew Krystal, MD, added, "Sleep plays a crucial role in neuropsychiatric diseases, and monitoring it in realistic, at-home settings is essential for understanding the full impact on patients.

The Inner Circle Acknowledges, Winston Richard Nara, MD, FCCP, DABSM, as a Top Pinnacle Professional for his contributions to the field of Pulmonology

Retrieved on: 
Wednesday, July 5, 2023
COPD, State University of New York, Society, University, Education, Internal medicine, Society of Critical Care Medicine, Physician, American Board of Internal Medicine, ABIM, State university system, Narcolepsy, Sleep apnea, Multimedia, Private practice, Pulmonology, Inner circle, Chronic obstructive pulmonary disease, American Board of Commissioners for Foreign Missions, Insomnia, COVID-19, MD, Patient, SUNY, Humanity, FCCP, Franciscan Health Indianapolis, Franciscan Health Mooresville, Diagnosis, Nursing care plan, Franciscan Health, American Board of Sleep Medicine, American College, Medical degree, Critical Care Medicine (journal), Dentistry, Pharmaceutical industry, Buffalo

GREENWOOD, Ind., July 5, 2023 /PRNewswire/ -- Prominently featured in The Inner Circle, Winston Richard Nara, MD, FCCP, DABSM, is acknowledged as a Top Pinnacle Professional for his contributions to the field of Pulmonology.

Key Points: 
  • GREENWOOD, Ind., July 5, 2023 /PRNewswire/ -- Prominently featured in The Inner Circle, Winston Richard Nara, MD, FCCP, DABSM, is acknowledged as a Top Pinnacle Professional for his contributions to the field of Pulmonology.
  • After relocating to the United States, the doctor completed his residency in internal medicine at Mount Sinai Queens Hospital in New York, NY.
  • He received fellowship training in pulmonary medicine, critical care, and sleep medicine at Buffalo's State University of New York (SUNY).
  • He is also a member of
    Aside from his professional pursuits, Dr. Nara supports his local community with the Indiana Habitat for Humanity.

China NMPA approves RareStone's pitolisant (Wakix) for the treatment of narcolepsy

Retrieved on: 
Tuesday, July 4, 2023
Narcolepsy, Cataplexy, EDS, Sleep paralysis, European Medicines Agency, Pitolisant, Approximation error, Government, EMA, Pharmacotherapy, Hypnagogia, Patient, Peking University, Diagnosis, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NMPA, Food, Disease, Pharmaceutical industry

SHANGHAI, July 4, 2023 /PRNewswire/ -- RareStone Group, a rare disease-focused company aiming to establish the first rare disease ecosystem in China, announced that on June 30, the Chinese National Medical Products Administration (NMPA)  approved pitolisant (Wakix) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. In China, pitolisant is the first approved innovative drug for narcolepsy and the only non-scheduled drug with narcolepsy indication.

Key Points: 
  • -- pitolisant is the first and only treatment drug approved in mainland China for narcolepsy.
  • Pitolisant has been listed in the Chinese guidelines for diagnosis and treatment for narcolepsy (2022) as a first-line treatment therapy.
  • According to , published by CHARD & Peking University, more than half of the narcolepsy patients in China have experienced misdiagnosis.
  • The fruitful and positive collaboration contributes a lot to bring this promising treatment drug to narcolepsy patients in China.

Avadel Pharmaceuticals to Host Commercial Day on June 29 for LUMRYZ™ (sodium oxybate) extended release for oral solution, CIII

Retrieved on: 
Thursday, June 22, 2023
Medicine, Cataplexy, Senior, Idiopathic hypersomnia, Internal medicine, Narcolepsy, EDS, Pediatrics, PLLC, Pulmonology, Webcast, Neurology, Commercial, Hospital, FDA, Developmental Medicine & Child Neurology, Pharmaceutical industry, Child

LUMRYZ was approved by the U.S. Food & Drug Administration (FDA) in May 2023 for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.

Key Points: 
  • LUMRYZ was approved by the U.S. Food & Drug Administration (FDA) in May 2023 for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.
  • Renowned sleep specialists will include:
    Dr. Anne Marie Morse is a board-certified neurologist and trained sleep medicine specialist.
  • Dr. Thomas Stern completed board certification in five different medical specialties including internal medicine, pediatrics, pulmonary medicine, critical care medicine and sleep medicine.
  • A replay of the webcast will be archived on Avadel’s website for 90 days following the event.

Zevra Therapeutics to be Added to Russell 2000® and Russell 3000® Indexes Effective June 26, 2023

Retrieved on: 
Tuesday, June 20, 2023
Growth, FTSE Russell, Narcolepsy, Acquisition, Therapy, Russell, Idiopathic hypersomnia, Niemann–Pick disease, Disease, Zebra

CELEBRATION, Fla., June 20, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, announced its expected addition to the broad-market Russell 3000® Index and small-cap Russell 2000® Index in accordance with the 2023 Russell indexes annual reconstitution.

Key Points: 
  • CELEBRATION, Fla., June 20, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, announced its expected addition to the broad-market Russell 3000® Index and small-cap Russell 2000® Index in accordance with the 2023 Russell indexes annual reconstitution.
  • Zevra anticipates that its inclusion in the Russell indexes will be effective after the U.S. market opens on Monday, June 26, 2023.
  • Membership in the U.S. all-cap Russell 3000 Index remains in place for one year and means automatic inclusion in the appropriate growth and value style indexes, which is the small-cap Russell 2000 Index for Zevra.
  • The 2023 index showed noteworthy growth in the health care industry, with a majority of the companies moving from small-cap to large-cap coming from the health care space.

Avadel Pharmaceuticals Announces Publication of Study Data in Advances in Therapy Highlighting Need for Once-at-Bedtime Oxybate Dosing for Narcolepsy

Retrieved on: 
Tuesday, June 13, 2023
Narcolepsy, Cataplexy, Doctor of Pharmacy, Senior, Gamma-Hydroxybutyric acid, Geisinger Medical Center, DCE, Discrete, Anxiety, Quality of life, Advances in Therapy, Patient, Somnolence, Neurology, Sodium, Developmental Medicine & Child Neurology, Pharmaceutical industry, Medical imaging, Child

The paper, titled “Clinician Preferences for Oxybate Treatment for Narcolepsy: Survey and Discrete Choice Experiment,” was published in Advances in Therapy and can be accessed here .

Key Points: 
  • The paper, titled “Clinician Preferences for Oxybate Treatment for Narcolepsy: Survey and Discrete Choice Experiment,” was published in Advances in Therapy and can be accessed here .
  • “Sodium oxybate has been recognized as a standard of care to treat narcolepsy for more than 20 years, but, until recently, has required two nighttime doses for sufficient therapy.
  • These data underscore the long unmet need for an oxybate treatment that does not require middle-of-the-night dosing.
  • “These data provide insight that the most important driver of oxybate choice is dosing and not sodium content.”

Koneksa Announces Partnership with Beacon Biosignals for Clinical Trial in Sleep and Neurologic Disorders Using EEG Biomarkers

Retrieved on: 
Tuesday, June 13, 2023
Technology, Medical Devices, Neurology, Science, Clinical Trials, Biotechnology, Wearables, Mobile Technology, Health, Other Science, Research, Software, Other Health, Mobile, Wireless, Hardware, Pharmaceutical, Data Management, Artificial Intelligence, Partnership, Drug development, Sleep, Observational study, Validity, Speech, Clinical trial, Joint Planning and Development Office, Narcolepsy, DSM-IV codes, Biomarker, Doctor of Philosophy, Patient, EEG, Algorithm, Entrepreneurship, Data, Obstructive sleep apnea, Medical imaging, Medical device, Pharmaceutical industry, MD, Beacon

Koneksa , the evidence-based digital biomarkers company, announced today a partnership with Beacon Biosignals , the leading computational neurodiagnostics company, to launch a clinical trial to investigate the integration of Beacon’s at-home electroencephalogram (EEG) into the Koneksa Neuroscience Solution Toolkit.

Key Points: 
  • Koneksa , the evidence-based digital biomarkers company, announced today a partnership with Beacon Biosignals , the leading computational neurodiagnostics company, to launch a clinical trial to investigate the integration of Beacon’s at-home electroencephalogram (EEG) into the Koneksa Neuroscience Solution Toolkit.
  • The LEARNS study will investigate the Koneksa neuroscience solution, as well as the Beacon Biosignals platform, applied to at-home EEG, compared with in-clinic polysomnography, in neurological and sleep disorders.
  • “The utilization of evidence-based digital biomarkers is the key to transforming healthcare,” said Chris Benko, CEO, Koneksa.
  • “Beacon’s mobile EEG will join more than a dozen digital health technologies that are already integrated into the Koneksa solution.