Sarcoidosis

First patient in the UK is dosed in the OATD-01 Phase 2 KITE study in pulmonary sarcoidosis

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星期五, 三月 22, 2024
PET/CT, Contract research organization, Biotechnology, Sarcoidosis, Disease, Patient safety, Patient, Tablet, Spirometry, Messenger, Quality of life, Biology, Royal, Fibrosis, Clinical trial, European, Arrest, Safety, Pharmacokinetics, MOC, Phase, KITE, FDA, FVC, ECG, Granuloma, Pharmaceutical industry

The world's first administration of the chitotriosidase 1 (CHIT1) inhibitor (or placebo) to patient took place at the Royal Infirmary in Edinburgh.

Key Points: 
  • The world's first administration of the chitotriosidase 1 (CHIT1) inhibitor (or placebo) to patient took place at the Royal Infirmary in Edinburgh.
  • As part of the trial, patients will take a daily fixed dose of 25 mg OATD-01 or placebo tablets for 12 weeks.
  • Patient safety will be monitored regularly through laboratory tests, neurological examinations and ECG and spirometry.
  • The study will involve approximately 20-30 centres in the US, the European Union, Norway and the UK.

aTyr Pharma Announces Fourth Quarter and Full Year 2023 Results and Provides Corporate Update

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星期四, 三月 14, 2024
PDT, Research, Conference, Interest, ATS, Biologics license application, Patient, SAN, Howard University, Knowledge, EAP, ATyr Pharma, EDT, NRP2, American Thoracic Society, Downstream, Investment, Clinical trial, Small RNA, Neuropilin, Myelocyte, Safety, Sarcoidosis, Immune system, FGF2, FGFR4, BLA, Pharmaceutical industry

SAN DIEGO, March 14, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced fourth quarter and full year 2023 results and provided a corporate update.

Key Points: 
  • SAN DIEGO, March 14, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced fourth quarter and full year 2023 results and provided a corporate update.
  • Based on current enrollment projections, the Company anticipates completing enrollment in the study in the second quarter of 2024.
  • Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information.
  • Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: http://investors.atyrpharma.com/events-and-webcasts .

Mallinckrodt Announces Journal Publication of Real-World Data on Acthar® Gel (repository corticotropin injection) to Treat Symptomatic Sarcoidosis in African American and Non-African American Patients

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星期一, 三月 11, 2024
African Americans, Conference, Sarcoidosis, Disease, Adrenocorticotropic hormone (medication), Inflammation, Patient, Research, Manuscript, Adrenocorticotropic hormone, Respiratory disease, Therapy, FDA, Food, Pharmaceutical industry

DUBLIN, March 11, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced the publication of findings from a retrospective chart review of Acthar® Gel (repository corticotropin injection) treatment outcomes for African American and non-African American patients with symptomatic sarcoidosis – including treatment patterns, co-medication use, and overall health outcomes.1 This analysis suggested that Acthar Gel treatment was associated with similar improvements in health outcomes, a reduction in symptoms, and reduced co-medication use in both African Americans and non-African Americans with symptomatic sarcoidosis.1 The manuscript was recently published online in Therapeutics & Clinical Risk Management.

Key Points: 
  • DUBLIN, March 11, 2024 /PRNewswire/ -- Mallinckrodt plc , a global specialty pharmaceutical company, today announced the publication of findings from a retrospective chart review of Acthar® Gel (repository corticotropin injection) treatment outcomes for African American and non-African American patients with symptomatic sarcoidosis – including treatment patterns, co-medication use, and overall health outcomes.1 This analysis suggested that Acthar Gel treatment was associated with similar improvements in health outcomes, a reduction in symptoms, and reduced co-medication use in both African Americans and non-African Americans with symptomatic sarcoidosis.1 The manuscript was recently published online in Therapeutics & Clinical Risk Management.
  • "The results of this retrospective medical chart review highlight unmet needs that exist for African American patients with symptomatic sarcoidosis, who are disproportionately affected by the disease, and reinforce Acthar Gel's potential to help improve health outcomes for appropriate patients,"1,4,5 said George Wan, Ph.D., M.P.H., Vice President, Evidence Generation and Data Sciences, Mallinckrodt.
  • "This research reflects Mallinckrodt's commitment to collecting real-world data on the relationship between patient characteristics, treatment patterns, and outcomes to support clinicians' treatment decisions and address disparities in symptomatic sarcoidosis care.
  • "1

Foundation for Sarcoidosis Research Partners to Host 30 Volunteer Patient Leaders at Unique In-Person Training at Cleveland Clinic

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星期五, 三月 1, 2024
Hospital, Cleveland Clinic, Caregiver, FSR, Sarcoidosis, Hope, Patient, Knowledge, Nursing

CHICAGO, March 01, 2024 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) is honored to host its inaugural in-person training of FSR-GSCA Volunteer Patient Leaders from around the United States at Cleveland Clinic, the FSR Global Sarcoidosis Clinic Alliance (FSR-GSCA) Founding Member.

Key Points: 
  • CHICAGO, March 01, 2024 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) is honored to host its inaugural in-person training of FSR-GSCA Volunteer Patient Leaders from around the United States at Cleveland Clinic, the FSR Global Sarcoidosis Clinic Alliance (FSR-GSCA) Founding Member.
  • This inaugural in-person training of FSR-GSCA Volunteer Patient Leaders has been structured to empower volunteer patient leaders with the skills, knowledge, and connections necessary to make a meaningful impact in the fight against sarcoidosis.
  • Cleveland Clinic is a proud member of the FSR Global Sarcoidosis Clinic Alliance and is honored to hold this training event in collaboration with FSR.
  • For media inquiries or more information about the FSR-GSCA Patient Volunteer Leaders’ Training Event, please contact [email protected] .

aTyr Pharma Announces Expanded Access Program (EAP) for EFZO-FIT™ Clinical Trial Participants

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星期三, 二月 21, 2024
SAN, Sarcoidosis, Scar, Small RNA, Pi, ATyr Pharma, FDA, Inflammation, Food, Scleroderma, Risk, Myelocyte, Health, Safety, Principal investigator, Expanded access, Neuropilin, Systemic scleroderma, ILD, OLE, Patient, Fibrosis, Clinical trial, EAP, Pharmaceutical industry

SAN DIEGO, Feb. 21, 2024 (GLOBE NEWSWIRE) --  aTyr Pharma, Inc. (Nasdaq: LIFE) (aTyr or the Company), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced plans to initiate an Individual Patient Expanded Access Program (EAP) for its lead therapeutic candidate, efzofitimod, for patients with pulmonary sarcoidosis. The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.

Key Points: 
  • Individual Patient EAP allows access to efzofitimod for patients who complete the Phase 3 EFZO-FIT™ study in pulmonary sarcoidosis.
  • Company initiating program based on blinded EFZO-FIT™ study investigator and patient participant feedback.
  • The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.
  • “We are pleased to make efzofitimod available to patients beyond the duration of the EFZO-FIT™ clinical trial through this Individual Patient EAP,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.

Tricha Shivas Promoted to Chief of Staff and Strategy at Foundation for Sarcoidosis Research

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星期四, 二月 15, 2024
Drug development, Hospital, Patient, Caregiver, Sarcoidosis, FSR, Management

CHICAGO, Feb. 15, 2024 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) proudly announces the promotion of Tricha Shivas to Chief of Staff and Strategy.

Key Points: 
  • CHICAGO, Feb. 15, 2024 (GLOBE NEWSWIRE) -- The Foundation for Sarcoidosis Research (FSR) proudly announces the promotion of Tricha Shivas to Chief of Staff and Strategy.
  • Ms. Shivas, who joined FSR in 2021 and has served FSR as the Vice President of Research and Strategic Partnerships and the Chief Strategy Officer, has played a pivotal role in the expansive growth of FSR over the last three years.
  • and caregivers committed to finding a cure and ensuring evidence-based, patient-centric care for those living with sarcoidosis.
  • “Since joining FSR three years ago, Tricha has proven to be an extraordinary leader filled with passion and expertise resulting in significant advancements for FSR,” said Mary McGowan, FSR’s Chief Executive Officer.

Autoimmune Disease and Pregnancy: ISB Study Challenges Prevailing Wisdom, Unveils Nuances

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星期三, 一月 31, 2024
Other Health, Research, Consumer, Healthcare Reform, Parenting, Science, Baby, Maternity, COVID-19, Other Science, Health, Diabetes, Public Policy, Government, Multimedia, Multiple sclerosis, Sarcoidosis, ISB, Mother, Health information management, Vasculitis, MD, Preterm birth, Inflammatory bowel disease, Antiphospholipid syndrome, Patient, Rheumatoid arthritis, Spondyloarthropathy, Cohort (statistics), Providence, Lupus, Hypertension, Pregnancy, Autoimmunity, Thought, Risk, Psoriatic arthritis, Psoriasis, Autoimmune disease, Gestational age, Birth control

View the full release here: https://www.businesswire.com/news/home/20240131144327/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240131144327/en/
    In a just-published study in the journal Lancet eClinical Health , researchers at the Institute for Systems Biology (ISB) and Providence showed nuanced pregnancy outcomes for pregnant individuals with autoimmune disease.
  • Each type of autoimmune disease is different, each person has their own medical history, and risk may change over the course of pregnancy.
  • This study highlights the importance of taking comorbidities into consideration,” said ISB Associate Professor Jennifer Hadlock, MD , who led the study.
  • “These results can help reshape discussions surrounding autoimmune disease and pregnancy, and bring fresh perspective to a traditionally understudied area in research,” Hadlock said.

Orphan designation: chimeric human-murine IgG1 kappa monoclonal antibody against TNF alfa Treatment of sarcoidosis, 25/07/2023 Positive

Retrieved on: 
星期日, 二月 4, 2024
TNF, Sarcoidosis, Immunoglobulin G, COMP, IRIS, EMA, European Commission, Patient, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - chimeric human-murine IgG1 kappa monoclonal antibody against TNF alfa
    - Intended use
    - Treatment of sarcoidosis
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2809
    - Date of designation
    - Sponsor
    FGK Representative Service GmbH
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

aTyr Pharma to Present Posters Highlighting Importance of Neuropilin-2 in Immune Regulation at Keystone Symposia on Myeloid Cell Diversity

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星期一, 一月 29, 2024
Patient, Medicine, Fibrosis, Faculty, Scar, SAN, NRP2, Scleroderma, University, Small RNA, Disease, Neuropilin, Nebraska Medicine, ATyr Pharma, Inflammation, Systemic scleroderma, Sarcoidosis, Myelocyte, Immune system, Pharmaceutical industry

SAN DIEGO, Jan. 29, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the company will present two posters at the Keystone Symposia on Myeloid Cell Diversity: From Fundamental Biology to Disease States, which is being held January 28 – 31, 2024, in Banff, Alberta, Canada.

Key Points: 
  • Findings further demonstrate that efzofitimod modulates myeloid cells via the neuropilin-2 (NRP2) receptor to promote a unique anti-inflammatory mechanism.
  • Role of NRP2 in immune system validated by activity of NRP2 blocking antibody in preclinical models.
  • aTyr Pharma, San Diego; Faculty of Health and Medical Sciences, Copenhagen, Denmark.
  • These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.

aTyr Pharma Announces Howard University President Emeritus Dr. Wayne A. I. Frederick as Advisor

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星期四, 一月 18, 2024
Mortality, ILD, Patient, Outline of health sciences, Wayne A. I. Frederick, Sarcoidosis, Research, Biology, Fibrosis, Associate, Scar, ACS, Dean (education), Howard University, SAN, Knowledge, University, National academy, Humana, Scleroderma, Business, Small RNA, American Surgical Association, Human, Disease, American College, Neuropilin, ATyr Pharma, Health, Inflammation, Organization, MD Anderson Cancer Center, Systemic scleroderma, College, Howard University Hospital, Abstract, Myelocyte, American Cancer Society, Pharmaceutical industry

Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.

Key Points: 
  • Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.
  • “We are honored to welcome a distinguished physician executive such as Dr. Frederick as an advisor to aTyr,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.
  • He is also the distinguished Charles R. Drew Professor of Surgery at the Howard University College of Medicine and a practicing cancer surgeon at Howard University Hospital.
  • Dr. Frederick earned a B.S., M.D., and completed his surgical residency training at Howard University Hospital.