Acute chest syndrome

AB Science: The clinical development of masitinib in sickle cell disease is among the 19 winning projects under the sixth call for “Hospital-Inuversity Research in health (RHU)”

Retrieved on: 
Lundi, novembre 27, 2023
SCD, Database, Polymerization, Recherche, Sickle cell disease, Partnership, Thalassemia, Risk, AP-HP, Death, Hemolytic anemia, Research, B cell, Basophil, VOC, FYN, Assistance, Mortality, Health, Disease, Acute chest syndrome, KIT, RBC, Mouse, Human, Life expectancy, Investment, Euronext, SCIENCE, Inflammation, Patent, Science, CELL, Public, Biomarker, Toxicity, RHU, Histamine, Child, Patient, APHP, Substance P, LYN, ACS, Pharmaceutical industry

THE CLINICAL DEVELOPMENT OF MASITINIB IN SICKLE CELL DISEASE, A HIGHLY PREVALENT GENETIC CONDITION, IS AMONG THE 19 WINNING PROJECTS UNDER THE SIXTH CALL FOR "HOSPITAL-UNIVERSITY RESEARCH IN HEALTH (RHU)".

Key Points: 
  • THE CLINICAL DEVELOPMENT OF MASITINIB IN SICKLE CELL DISEASE, A HIGHLY PREVALENT GENETIC CONDITION, IS AMONG THE 19 WINNING PROJECTS UNDER THE SIXTH CALL FOR "HOSPITAL-UNIVERSITY RESEARCH IN HEALTH (RHU)".
  • A new patent has been filed, which, if granted, will extend the international protection of masitinib in sickle cell disease until 2040.
  • Current treatment options such as hydroxycarbamide, chronic transfusion or anti-P-selectin antibodies, do not fully prevent life-threatening acute and chronic complications of sickle cell disease.
  • There is a significant medical need to prevent the acute and chronic complications of sickle cell disease.

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events

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Samedi, décembre 9, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Adolescence, Nasal congestion, Safety, Food, Hemolytic anemia, Sickle cell disease, ASH, Pain, Priapism, Acute myeloid leukemia, Patient, Disease, Acute chest syndrome, Hospital, Quality of life (healthcare), Fatigue, HB, Leukemia, Busulfan, Division, VOE, RAF Sullom Voe, Hemolysis, Myelodysplastic syndrome, Exposition, University, Diagnosis, Society, Quality of life, Biology, Blood transfusion, Bilirubin, Comorbidity, Pharmaceutical industry, Medical imaging, Anemia, Hematology

The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Key Points: 
  • The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.
  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field.
  • As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.
  • In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia.

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
Vendredi, décembre 8, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Red blood cell, Sickle cell disease, Patient, Anemia, Quality of life, Bangladesh Technical Education Board, Qualification, Webcast, Priority review, Lung, Vasculitis, Food, Incidence, Pain, Priapism, Acute myeloid leukemia, Bluebird, PIN, Genetic disorder, Regenerative medicine, Division, Ronas Voe, DSM-IV codes, Hematology, Vaso-occlusive crisis, Courage, Adolescence, Hemolytic anemia, Stomatitis, Cellular, Febrile neutropenia, History, Leukopenia, Thrombocytopenia, Neutropenia, University, QTC, Life, NHLBI, Bone marrow, Research, HBA, Bluebird bio, Telephone, Disease, Acute chest syndrome, Caregiver, NIH, MD, Blood transfusion, Pharmaceutical industry, Vaccine, Online shopping

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.

bluebird bio Details Plans for the Commercial Launch of LYFGENIA™ Gene Therapy for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
Vendredi, décembre 8, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Cardiology, Bone marrow, Adolescence, Medicaid, Research, Acquisition, Sickle cell disease, Stomatitis, Incidence, Pain, Priapism, Acute myeloid leukemia, Bluebird, Disease, Risk, Acute chest syndrome, Febrile neutropenia, History, Leukopenia, Patient, Thrombocytopenia, FDA, VOE, QTC, Voe Minas Gerais, Stroke, Caregiver, Neutropenia, Death, Anemia, Quality of life, Qualification, Life, Blood transfusion, Education, Pharmaceutical industry, Nursing, Vaccine

“Our approach is built on the principle that timely, equitable access to gene therapy is imperative for people living with sickle cell disease,” said Tom Klima, chief commercial & operating officer, bluebird bio.

Key Points: 
  • “Our approach is built on the principle that timely, equitable access to gene therapy is imperative for people living with sickle cell disease,” said Tom Klima, chief commercial & operating officer, bluebird bio.
  • The burden of sickle cell disease is immense and can impact every aspect of life for patients and their loved ones.
  • Fifty to 60% of adults living with sickle cell disease have end organ damage, and one in four people living with sickle cell disease have a stroke by age 45.
  • “The FDA approval of a sickle cell disease gene therapy may be a game changer,” said Dr. Shantel Herbert-Magee, Chief Medical Director, Louisiana Medicaid.

Sickle Cell Disease Partnership Calls for Passage of Key Legislation

Retrieved on: 
Jeudi, septembre 22, 2022
African Americans, Health, Health literacy, Child, Oxygen, Senior advisor, Acute chest syndrome, Disease, Legislation, Stroke, Infection, Patient, Mental health, Federation, Travel, Medicaid, Senate, SCD, Pain, Sickle cell disease, House, National academy, Executive branch of the government of Honduras, National Academy of Medicine, Research, Executive (government), Congress, Health insurance, Pharmaceutical industry, Vaccine

WASHINGTON, Sept. 22, 2022 /PRNewswire/ -- As the nation commemorates National Sickle Cell Awareness Month, the Sickle Cell Disease Partnership is calling on Congress to pass three pieces of legislation that will increase access to treatment and improve the lives of the 100,000 plus Americans living with the disease. Together, the Sickle Cell Disease Comprehensive Care Act (H.R. 6216/S.3389), Sickle Cell Care Expansion Act (H.R. 7177/S.4425) and the newly introduced Treatment Centers Act of 2022 (H.R.8855/S.4866) will boost funding for research and increase access to life-changing treatment.

Key Points: 
  • During National Sickle Cell Awareness Month, Partnership Calls on Policymakers to Improve Health Access, Treatment
    WASHINGTON, Sept. 22, 2022 /PRNewswire/ --As the nation commemoratesNational Sickle Cell Awareness Month, the Sickle Cell Disease Partnership is calling on Congress to pass three pieces of legislation that will increase access to treatment and improve the lives of the 100,000 plus Americans living with the disease.
  • The Sickle Cell Disease Treatment Centers Act of 2022 (H.R.8855/S.4866) establishes a nationwide network of more than 120 Sickle Cell Disease Treatment Centers based on a hub-and-spoke framework and provides support for 100 community-based organizations.
  • Sickle Cell Disease is a rare, genetic blood disorder that primarily affects African American individuals.Sickle Cell Disease is inherited when a child receives two sickle cell genesone from each parent.
  • The Sickle Cell Disease Partnership is a multi-sectorcollaborationofhealth carestakeholders committed to advancingactionablefederal health carepoliciesthat will improve the lives of patients living with Sickle Cell Disease.

GBT Supports the Sickle Cell Disease Treatment Centers Act of 2022

Retrieved on: 
Jeudi, septembre 15, 2022
Center, Oxygen, Acute chest syndrome, Hemolysis, FDA, National Heart, Lung, and Blood Institute, Blood, Polymerization, Legislation, United, Population, Volatile organic compound, Health, Sickle, Stroke, End organ damage, Patient, Sickle cell disease, GBT, Anti-transglutaminase antibodies, Lung, Physician, CEO, The Council, SCD, Data collection, HBS, Centers for Disease Control and Prevention, Life expectancy, Tissue, Caregiver, NASDAQ, European Medicines Agency, Statistics, Research, Capillary, University, SAN, GLOBE, United States $1 Coin Act of 1997, Education, Disease, Council, Anemia, Pharmaceutical industry, Medical device

SOUTH SAN FRANCISCO, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc.(GBT) (NASDAQ: GBT) announced its support for the Sickle Cell DiseaseTreatment Centers Act of 2022, which was introduced today and, if passed in its current form, would authorize for appropriations of $535 million in annual funding to support sickle cell disease (SCD) treatment, research and education.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc.(GBT) (NASDAQ: GBT) announced its support for the Sickle Cell DiseaseTreatment Centers Act of 2022, which was introduced today and, if passed in its current form, would authorize for appropriations of $535 million in annual funding to support sickle cell disease (SCD) treatment, research and education.
  • The key elements of the Sickle Cell DiseaseTreatment Centers Act of 2022 are based on a vision developed by a diverse group of stakeholders representing individuals living with sickle cell disease, caregivers, advocates, community-based organizations, physicians, and payers.
  • The introduction and passing of this act have the potential to dramatically improve the lives of individuals living with sickle cell disease across the United States, said Biree Andemariam, M.D., director of the New England Sickle Cell Institute at the University of Connecticut and the chairperson of The Council for Sickle Cell Disease Health Equity.
  • The establishment and funding of more than 100 treatment centers would ensure far greater access to quality expert care for those living with sickle cell disease around the country, filling a longstanding inequity and largely ignored gap in the current healthcare landscape for the sickle cell disease community.

Folia Health Technology Platform Used in First-of-its-Kind Real World Evidence Study for Sickle Cell Disease

Retrieved on: 
Jeudi, septembre 15, 2022
Infection, Hip, Center, Sickle cell disease, Acute chest syndrome, Voxelotor, Senior, High reliability organization, Stroke, End organ damage, Patient, GBT, ASCEND, CEO, SCD, Data collection, Degenerative disease, Blood, Anti-transglutaminase antibodies, Doctor of Pharmacy, Centers for Disease Control and Prevention, Founder, Caregiver, Statistics, Research, GLOBE, Health, Splenectomy, Disease, Individual, Pharmaceutical industry, Medical imaging

BOSTON, Sept. 15, 2022 (GLOBE NEWSWIRE) -- Today, Folia Health announced the initiation of the ASCEND Study, a first-of-its-kind, at-home observational real-world evidence study in which people living with sickle cell disease (SCD) will use Folias innovative technology platform to track their individual changes in symptoms and treatment use.

Key Points: 
  • BOSTON, Sept. 15, 2022 (GLOBE NEWSWIRE) -- Today, Folia Health announced the initiation of the ASCEND Study, a first-of-its-kind, at-home observational real-world evidence study in which people living with sickle cell disease (SCD) will use Folias innovative technology platform to track their individual changes in symptoms and treatment use.
  • The ASCEND Study, which is currently enrolling patients, is being conducted by Folia Health with funding from Global Blood Therapeutics, Inc. (GBT).
  • The ASCEND study will utilize the Folia mobile app or desktop platform to capture this evidence directly from patients in real time.
  • We hope that using the Folia Health platform in the ASCEND Study will deliver new insights by enabling sickle cell disease patients to track their experiences and participate in meaningful research from their home.

The Foundation for Sickle Cell Disease Research Hosts Virtual Symposium Highlighting the Applications of Machine Learning in Sickle Cell

Retrieved on: 
Jeudi, septembre 15, 2022
University of Tennessee Health Science Center, Adoption, VP, Therapy, Symposium, Machine learning, Center, CME, UTHSC, Manifest and latent functions and dysfunctions, Learning, Social media, COO, Patient, Sickle cell disease, Intelligence, Physician, SCD, Technology, Solution, Informatics, Foundation, Research, Acute chest syndrome, University, Health informatics, Science, Medical device, Pharmaceutical industry

FORT LAUDERDALE, Fla., Sept. 15, 2022 /PRNewswire/ -- On September 22nd, during Sickle Cell Awareness Month, The Foundation for Sickle Cell Disease Research (FSCDR), the US's first outpatient center exclusively dedicated to the treatment of and innovative research for SCD, will host a one-day virtual symposium on featured topic: the Applications of Machine Learning in Sickle Cell.

Key Points: 
  • FORT LAUDERDALE, Fla., Sept. 15, 2022 /PRNewswire/ -- On September 22nd, during Sickle Cell Awareness Month, The Foundation for Sickle Cell Disease Research (FSCDR), the US's first outpatient center exclusively dedicated to the treatment of and innovative research for SCD, will host a one-day virtual symposium on featured topic: the Applications of Machine Learning in Sickle Cell .
  • They all share the common goal of finding better treatment, and ultimately, a cure for sickle cell disease by exploring and learning about the future of artificial intelligence and technology in healthcare.
  • Sickle cell warriors and their caretakers are invited to participate in the symposium at no cost.
  • We are hopeful that machine learning is one of many tools that offer life-saving solutions to patients in the future."

Sickle Cell Disease Treatment Global Market Report 2022: 16.2% Annual Growth Driven by Key Players AstraZeneca, Baxter, Bristol-Myers Squibb & Others - ResearchAndMarkets.com

Retrieved on: 
Lundi, septembre 12, 2022
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Sickle cell disease, COVID-19, Acute chest syndrome, National Heart, Lung, and Blood Institute, NHLBI, Blood, RBC, Life, VOC, USFDA, Patient, Lung, SCD, Pain, Bone marrow, Cell, Research, DNA, Medicine, R, Awareness, Anemia, CAGR, Pharmaceutical industry, Growth

The "Sickle Cell Disease Treatment Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Sickle Cell Disease Treatment Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.
  • The global sickle cell disease treatment market size reached US$ 2.6 Billion in 2021.
  • Sickle cell disease (SCD) is a genetic red blood cell (RBC) disorder that affects hemoglobin, a protein that carries oxygen throughout the body.
  • The publisher provides an analysis of the key trends in each sub-segment of the global sickle cell disease treatment market report, along with forecasts at the global, regional and country level from 2022-2027.

The GBT Foundation Awards $250,000 in ACE Grants to Support Sickle Cell Disease Community-Based Organizations

Retrieved on: 
Mercredi, juillet 13, 2022
Well, Mindfulness, National Heart, Lung, and Blood Institute, Education, Homeland Security Grant Program, Centers for Disease Control and Prevention, United, Patient, SAN, Oxygen, Sickle cell disease, Lung, Capillary, Tissue, Health equity, Anemia, Transition, Therapy, Organization, GLOBE, Excellent Care for All Act, Volatile organic compound, Disease, Criterion, ACE, Sickle Cell Disease Association of America, Center, Adult, Blood, Health, GBT, Polymerization, FDA, SCD, Hemolysis, Empowerment, Access, European Medicines Agency, Statistics, Government, Sickle, Stroke, HBS, Acute chest syndrome, End organ damage, Head, Caregiver, Andrew Kohut, ACCEL, Pharmaceutical industry, Medicine

Through the ACE Grant Program, The GBT Foundation plans to fund up to $250,000 per year to support initiatives dedicated to advancing health equity and enhancing education, empowerment, and access to care for people living with sickle cell disease (SCD).

Key Points: 
  • Through the ACE Grant Program, The GBT Foundation plans to fund up to $250,000 per year to support initiatives dedicated to advancing health equity and enhancing education, empowerment, and access to care for people living with sickle cell disease (SCD).
  • CBOs can play an important role as local resources for sickle cell disease patients and caregivers as they navigate their healthcare journey.
  • Childrens Sickle Cell Foundation, Inc. (Pittsburgh, Pennsylvania) Living Well with Sickle Cell Family Leadership Program to educate sickle cell family leaders about SCD, care, treatment, and their role in advocacy.
  • Sickle Cell Association of South Louisiana (Lafayette, Louisiana) KNEAUX Sickle Program to support resources for teens transitioning from pediatric to adult SCD healthcare providers.