Rare

X4 Pharmaceuticals Announces Appointment of Industry Veteran R. Keith Woods to Board of Directors

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水曜日, 10月 18, 2023
Rare, Eisai, Senior, Immune system, Marketing, Amgen, Retirement, Roche, Florida State University, Alexion Pharmaceuticals, Pharmaceutical industry, Management

BOSTON, Oct. 18, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the appointment of R. Keith Woods as an independent director to the company’s Board of Directors.

Key Points: 
  • BOSTON, Oct. 18, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the appointment of R. Keith Woods as an independent director to the company’s Board of Directors.
  • “We are thrilled to welcome Keith to the X4 Board of Directors,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • Following his retirement from argenx, Mr. Woods continues to serve as an advisor to the argenx Board of Directors.
  • Mr. Woods commented on his appointment: “It is a privilege to join the X4 Board at this exciting time, given the recent U.S. regulatory submission of the company’s first drug candidate and potential upcoming U.S. commercial launch.

Sage Therapeutics Announces U.S. Food and Drug Administration Granted SAGE-718 Orphan Drug Designation for the Treatment of Huntington’s Disease

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水曜日, 10月 18, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, FDA, Clinical Trials, AD, Orphan Drug Act of 1983, PD, Food, MCI, Rare, DSM-IV codes, HD, European Medicines Agency, Cognitive deficit, Deficit spending, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ODD, Disease, Clinical trial, Marketing, Therapy, NMDA, Patient, Plasma protein binding, Pharmaceutical industry

Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD).

Key Points: 
  • Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD).
  • Cognitive impairment is one of the most underrecognized aspects of this disease,” said Laura Gault, M.D., Ph.D., Chief Medical Officer, Sage Therapeutics.
  • The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.
  • SAGE-718 previously received Fast Track Designation from the FDA for HD, and orphan drug designation for HD by the European Medicines Agency.

GC Genome to Present the Latest Lung Cancer Diagnostic Tool at ESMO 2023

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金曜日, 10月 20, 2023
EAP, AUC, ESMO, Oncology, Lung cancer, Congress, European Society for Medical Oncology, CAP, Liver, Research, Access, Lung, Genomics, Methylation, Rare, Genome, Cancer, Infant, GC, Deep learning, Patient, Diagnosis, Medical imaging

YONGIN, South Korea, Oct. 20, 2023 /PRNewswire/ -- GC Genome Corporation, a leading diagnostics company, today announced that it will present data showcasing deep learning algorithm utilizing methylation markers that can identify lung cancer at the European Society for Medical Oncology (ESMO) 2023 Congress, taking place from October 20-24 in Madrid, Spain. The new data showcases GC Genome's ongoing dedication to assisting individuals battling cancer.

Key Points: 
  • YONGIN, South Korea, Oct. 20, 2023 /PRNewswire/ -- GC Genome Corporation, a leading diagnostics company, today announced that it will present data showcasing deep learning algorithm utilizing methylation markers that can identify lung cancer at the European Society for Medical Oncology (ESMO) 2023 Congress, taking place from October 20-24 in Madrid, Spain.
  • Using a targeted EM-seq panel, the lung cancer detection performance achieved an accuracy of 81.5% and an AUC of 0.87.
  • Moreover, the serial dilution experiments have demonstrated the capability to detect lung cancer with a sensitivity of 80% down to a tumor fraction of 0.1%, offering a highly sensitive and accurate diagnostic tool for lung cancer.
  • Established in 2013 as a GC company, GC Genome operates a CAP-accredited laboratory and places the utmost emphasis on R&D.

Global Genes and the Rare Disease Diversity Coalition Expand Effort to Accelerate Diagnosis of Rare Disease in Underserved Communities

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木曜日, 10月 19, 2023
Genetic counseling, DSM-IV codes, Genetic testing, Health, Rare, AL, Rare disease, Navigation, Gene, Community, Personal Genetics Education Project, Quality of life, Disease, Caregiver, Partnership, Your Family Tree, Teaching, Global Genes, Patient, Black Women's Health Imperative, Nursing, Medical device, Vaccine, Delays, Diagnosis

ALISO VIEJO, Calif., Oct. 19, 2023 /PRNewswire-PRWeb/ -- Global Genes, a leading rare disease patient advocacy organization, and the Rare Disease Diversity Coalition (RDDC) today launches the second phase of their Know Your Family History initiative, which promotes more accurate, timely, and inclusive diagnoses within underserved communities.

Key Points: 
  • ALISO VIEJO, Calif., Oct. 19, 2023 /PRNewswire-PRWeb/ -- Global Genes, a leading rare disease patient advocacy organization, and the Rare Disease Diversity Coalition (RDDC) today launches the second phase of their Know Your Family History initiative, which promotes more accurate, timely, and inclusive diagnoses within underserved communities.
  • "By working with community health workers who are embedded in the communities they serve, this initiative will go a long way in increasing awareness of rare diseases at the grassroots level and help accelerate the path to a diagnosis and treatment."
  • - Shruti Mitkus, Global Genes
    Know Your Family History is part of a multifaceted partnership between Global Genes and the RDDC.
  • RDDC was launched by Black Women's Health Imperative to address the challenges faced by underserved populations with rare diseases and to reduce health disparities.

EveryLife Foundation for Rare Diseases Announces Search for CEO to Lead New Era of Growth

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木曜日, 10月 19, 2023
Rare, Culture, Growth, Interest, Disease, Policy, Education, Public policy, Letter, Multimedia, Management

The Board of Directors decided to transition the Foundation's top post from an executive director to a CEO to focus intently on strategy and fundraising.

Key Points: 
  • The Board of Directors decided to transition the Foundation's top post from an executive director to a CEO to focus intently on strategy and fundraising.
  • The incoming CEO will report directly to the board and play a pivotal role in realizing the strategic vision for this era of growth.
  • The incoming CEO will spearhead the development of fresh strategies and fundraising initiatives, paving the way for sustained progress."
  • The Foundation is in pursuit of a CEO who blends inspirational leadership with sophisticated management acumen.

Daewoong Pharmaceutical to Participates in CPHI Worldwide 2023, Showcasing Own Novel Drug in the Global Market

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木曜日, 10月 19, 2023
Gastroenterology, Exhibition, Rare, Booth, Hall, GERD, Fira (Barcelona Metro), Partnership, Daewoong Pharmaceutical, Autoimmunity, Pain management, Pharmaceutical industry

SEOUL, South Korea, Oct. 19, 2023 /PRNewswire/ -- Daewoong Pharmaceutical is thrilled to announce its participation in CPHI Worldwide 2023, one of the world's premier pharmaceutical industry exhibitions.

Key Points: 
  • SEOUL, South Korea, Oct. 19, 2023 /PRNewswire/ -- Daewoong Pharmaceutical is thrilled to announce its participation in CPHI Worldwide 2023, one of the world's premier pharmaceutical industry exhibitions.
  • The event is scheduled to take place from the 24th to the 26th of October at Fira Barcelona Gran Via, Spain.
  • Especially, Daewoong Pharmaceutical will accelerate to build partnership with global partner for their own novel drug such as GERD treatment (ingredient: Fexuprazan) and Diabetes treatment (ingredient: Enavogliflozin).
  • Additionally, the company has entered into technology export agreements with 18 countries, including China, further solidifying its global reach.

Daewoong Pharmaceutical to Participates in CPHI Worldwide 2023, Showcasing Own Novel Drug in the Global Market

Retrieved on: 
木曜日, 10月 19, 2023
Gastroenterology, Exhibition, Rare, Booth, Hall, GERD, Fira (Barcelona Metro), Partnership, Daewoong Pharmaceutical, Autoimmunity, Pain management, Pharmaceutical industry

SEOUL, South Korea, Oct. 19, 2023 /PRNewswire/ -- Daewoong Pharmaceutical is thrilled to announce its participation in CPHI Worldwide 2023, one of the world's premier pharmaceutical industry exhibitions.

Key Points: 
  • SEOUL, South Korea, Oct. 19, 2023 /PRNewswire/ -- Daewoong Pharmaceutical is thrilled to announce its participation in CPHI Worldwide 2023, one of the world's premier pharmaceutical industry exhibitions.
  • The event is scheduled to take place from the 24th to the 26th of October at Fira Barcelona Gran Via, Spain.
  • Especially, Daewoong Pharmaceutical will accelerate to build partnership with global partner for their own novel drug such as GERD treatment (ingredient: Fexuprazan) and Diabetes treatment (ingredient: Enavogliflozin).
  • Additionally, the company has entered into technology export agreements with 18 countries, including China, further solidifying its global reach.

Inhibrx Announces Participation in Upcoming Scientific Conferences

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水曜日, 10月 18, 2023
SAN, Rare, Ewing sarcoma, Agonist, Therapy, Nightclub

SAN DIEGO, Oct. 18, 2023 /PRNewswire/ -- Inhibrx, Inc. (Nasdaq: INBX), a clinical-stage biopharmaceutical company dedicated to the development of therapeutics for oncology and rare diseases, today announced it will be presenting at the following upcoming scientific conferences:

Key Points: 
  • SAN DIEGO, Oct. 18, 2023 /PRNewswire/ -- Inhibrx, Inc. (Nasdaq: INBX), a clinical-stage biopharmaceutical company dedicated to the development of therapeutics for oncology and rare diseases, today announced it will be presenting at the following upcoming scientific conferences:
    Title: The Tetravalent Death Receptor 5 Agonist INBRX-109 Combined With Chemotherapy in Ewing Sarcoma: Preliminary Data From A Phase 1 Study
    Location: The Liffey Convention Centre in Dublin, Ireland
    Trial-in-progress poster presentation on Saturday, November 4th, 2023 from 9 AM – 8:30 PM Pacific Standard Time
    Trial-in-progress poster presentation on Saturday, November 4th, 2023 from 9 AM – 8:30 PM Pacific Standard Time
    Location: San Diego Convention Center in San Diego, CA - Hall A
    The posters will be accessible through a link on the investors' section of Inhibrx's website at https://inhibrx.investorroom.com/events-and-presentations upon commencement of each respective presentation.

JPA HEALTH EXPANDS LIFE SCIENCES DIVISION WITH ACQUISITION OF TRUE NORTH SOLUTIONS, INC.

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水曜日, 10月 18, 2023
Cloud, Data analysis, EVP, KPI-driven code analysis, Rare, Government, Data, Health, Acquisition, CNS, JPA, Google Cloud, Natural language processing, Marketing, True north, Infection, AI, Acceleration, Pharmaceutical industry, Industrial design

WASHINGTON, Oct. 18, 2023 /PRNewswire/ -- JPA Health, a global full-service, integrated agency, today announced the acquisition of True North Solutions, Inc., a consulting firm based in Cambridge, Mass. True North specializes in commercial, clinical, and medical affairs, with expertise in digital acceleration and customized predictive analytics, as well as solutions powered by AI and natural language processing. This acquisition, the first for JPA Health, will further expand JPA's strategic offerings providing end-to-end integrated medical affairs, clinical development, commercial strategy, creative marketing, public relations and patient advocacy solutions to clients.

Key Points: 
  • This acquisition, the first for JPA Health, will further expand JPA's strategic offerings providing end-to-end integrated medical affairs, clinical development, commercial strategy, creative marketing, public relations and patient advocacy solutions to clients.
  • "Here at JPA Health, we utilize cutting-edge data and analytics technology to uncover valuable insights that fuel our success," said Carrie Jones, CEO of JPA Health.
  • We extend a warm welcome to the entire True North team, individuals who share our passion for helping people live healthier lives."
  • All True North employees, including Baughman, will join JPA Health.

Rezolute Receives Priority Medicines (PRIME) Eligibility from European Medicines Agency for Enhanced Regulatory Support of RZ358 in Congenital Hyperinsulinism

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火曜日, 10月 17, 2023
RIZE, Therapy, JD, European Medicines Agency, Rezolution Pictures, Disease, EMA, HI, Rare, Congenital hyperinsulinism, Clinical trial, PRIME, Hypoglycemia, Patient, Pharmaceutical industry

REDWOOD CITY, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) eligibility to RZ358 for the treatment of congenital hyperinsulinism (HI).

Key Points: 
  • REDWOOD CITY, Calif., Oct. 17, 2023 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) eligibility to RZ358 for the treatment of congenital hyperinsulinism (HI).
  • PRIME eligibility is granted by EMA to drug candidates that target an unmet medical need and show potential benefit for patients based on clinical trial results.
  • The PRIME initiative was created to provide proactive and enhanced support to developers of promising medicines to optimize development plans and accelerate evaluation with the goal of having new therapies reach patients faster.
  • “We are excited to receive PRIME eligibility for RZ358, especially as we plan to initiate our Phase 3 study for RZ358 this quarter,” remarked Susan Stewart, JD, Chief Regulatory Officer at Rezolute.