Bone pain

Freeline Presents Positive New Data from Phase 1/2 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Late-Breaking Oral Presentation at ASGCT 27th Annual Meeting

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星期四, 五月 9, 2024
FDA, Bone marrow, Mutation, Cell, Risk, Blood, News, Research, ERT, White blood cell, Glycogen storage disease, SRT, ALT, GBA1, White, European Medicines Agency, Brain, Enzyme replacement therapy, Regenerative Medicine Advanced Therapy, Therapy, Plasma, Patient, Mouse, Safety, Tissue, Glucocerebrosidase, Substantia nigra, Lysosome, Neuropsychological test, AAV, Dementia, PRIME, Fatigue, EMA, Drug development, Bone pain, Gaucher's disease, FACIT, Disease, Liver, Gene, Society, Standard of care, Annual general meeting, Pharmaceutical industry

LONDON, May 09, 2024 (GLOBE NEWSWIRE) --  Freeline Therapeutics today announced new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, showing substantial reductions in glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response, in patients with persistently high levels despite years of treatment with currently approved therapies, as well as early signs of clinical improvements in bone marrow burden and fatigue. FLT201 continues to demonstrate a favorable safety and tolerability profile. These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.

Key Points: 
  • These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.
  • Reductions in lyso-Gb1 levels in the blood are highly correlated with substrate reduction in disease-affected tissues and positive clinical outcomes in Gaucher disease.
  • Currently approved treatments have made a significant difference for people with Gaucher disease, but there is not an existing cure.
  • Both the late-breaking oral presentation and the poster presentation are now available on the News & Events section of Freeline’s website.

Freeline to Present New Data on its Gaucher and Parkinson’s Disease Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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星期三, 四月 24, 2024
Bone pain, Congress, Gaucher's disease, Glucocerebrosidase, News, Abstract, Cell, Fracture, Patient, Gene, Society, Annual general meeting, Therapy, Pharmaceutical industry

LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.

Key Points: 
  • LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.
  • “FLT201, our investigational gene therapy for Gaucher disease, has the potential to deliver better efficacy with a one-time treatment, and we are excited to present new clinical data from our ongoing Phase 1/2 trial in a late-breaking oral presentation at ASGCT.
  • We will also be sharing new data from our Parkinson’s disease research program, which builds on our work in Gaucher disease by leveraging the enhanced stability of our rationally engineered GCase enzyme.
  • The presentation materials will be available on the News & Events section of Freeline’s website following presentation at the ASGCT Annual Meeting.

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

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星期二, 四月 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, B cell, Gaucher's disease, Cell, CD34, Bone marrow, Bleeding, Disease, Seizure, Bruise, Patient, Committee, Knowledge, Fracture, Spleen, Regulation, Fatigue, Autotransplantation, EMA, IRIS, Human, Life expectancy, Eye, Bone pain, Anemia, European, COMP, Sponsor, European Commission, Safety, PPD, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

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星期二, 四月 9, 2024
Eurostat, Diagnosis, Civil service commission, Radiation, Brain, Ornithine decarboxylase, Enzyme, Growth, Weakness, Survival, Cell, Neuroblastoma, Disease, Patient, Tablet, Committee, Knowledge, Abdomen, Regulation, Child, Cancer, EMA, IRIS, Bone pain, Mouth, European, Polyamine, Fever, COMP, Sponsor, European Commission, Spine, Safety, Marketing, Limited, Medicine, Pharmaceutical industry

Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

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Orphan designation: Eflornithine Treatment of neuroblastoma, 27/09/2011 Withdrawn

PANTHERx®Rare Selected by Edenbridge Pharmaceuticals as Specialty Pharmacy for Yargesa® (miglustat 100 mg capsules)

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星期一, 四月 1, 2024
Disease, Capsule, Patient, Pharmacy, Bone pain, Hepatomegaly, Enzyme replacement therapy, Anemia, Gaucher, Thrombocytopenia, Liver, Spleen, Pharmaceutical industry

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.

Key Points: 
  • Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.
  • Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.
  • This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.
  • "We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease.

Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

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星期二, 一月 2, 2024
Spleen, Patient, Liver, Medical Subject Headings, Marketing, Disease, INN, Metabolism, Fatigue, Heartburn, Fracture, ATC, Gaucher's disease, Capsule, International, Enzyme replacement therapy, Bone pain, Language, Mouth, CYP2D6, European Medicines Agency, Indigestion, EMS, Select, Instant messaging, Bruise, Safety, Anatomy, Anemia, IIA, Medical device

Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

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Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

Human medicines European public assessment report (EPAR): Ebvallo, tabelecleucel, Date of authorisation: 16/12/2022, Revision: 3, Status: Authorised

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星期二, 一月 2, 2024
Medical Subject Headings, Diarrhea, Marketing, Agency, INN, Fever, Immune system, Fatigue, Constipation, Infection, Lymph, Hypotension, Virus, Bone marrow, Graft-versus-host disease, International, PTLD, Nasal congestion, Disease, Bone pain, Nausea, Language, Lymphoproliferative disorders, Cancer, Liver, Hyponatremia, Rash, Neutrophil, Appetite, Observation, European Medicines Agency, Transplant, Hypoxia, Select, Cell, Patient, Human, Safety, Dehydration, Anemia, Alkaline phosphatase, IIA, Epstein–Barr virus, Medical device

Human medicines European public assessment report (EPAR): Ebvallo, tabelecleucel, Date of authorisation: 16/12/2022, Revision: 3, Status: Authorised

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Human medicines European public assessment report (EPAR): Ebvallo, tabelecleucel, Date of authorisation: 16/12/2022, Revision: 3, Status: Authorised

Human medicines European public assessment report (EPAR): Jakavi, ruxolitinib, Date of authorisation: 23/08/2012, Revision: 29, Status: Authorised

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星期二, 一月 2, 2024
Janus, Liver, Marketing, PV, INN, Hypertension, Graft-versus-host disease, Polycythemia, Hydroxycarbamide, Infection, Transplant, Splenomegaly, Bruise, Polycythemia vera, Bacteria, Anatomy, IIA, Spleen, Fever, Hypercholesterolemia, Primary myelofibrosis, ATC, Bleeding, Disease, Ageing, JAK, Neutrophil, Blood, System, Disorder, Weight gain, Enzyme, Safety, Anemia, Sepsis, Medical Subject Headings, Agency, B cell, International, Bone pain, Neutropenia, Language, Mouth, Dizziness, Thrombocytopenia, Pancreatic serous cystadenoma, Hypertriglyceridemia, Tablet, Select, Patient, Medicine, Phlebotomy, Hormone, Woman, Weight loss, Headache, Cytomegalovirus, European Medicines Agency, Growth, Janus kinase, Urinary tract infection, Medical device

Human medicines European public assessment report (EPAR): Jakavi, ruxolitinib, Date of authorisation: 23/08/2012, Revision: 29, Status: Authorised

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Human medicines European public assessment report (EPAR): Jakavi, ruxolitinib, Date of authorisation: 23/08/2012, Revision: 29, Status: Authorised

Metastatic Castration-Resistant Prostate Cancer Market is Predicted to Exhibit Remarkable Growth at a CAGR of 7.5% During the Study Period (2019-2032), Estimates DelveInsight

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星期三, 十二月 20, 2023
GT0918, Niraparib, Phase, Ipsen, CRPC, Prognosis, Daiichi Sankyo, PSMA, BRCA, FDA, Spacecraft, Gesellschaft mit beschränkter Haftung, DNA, EU4, Cancer, Novartis, ICIS, Abiraterone acetate, Ageing, Urination, PSA, Metastasis, CYP17, Survival rate, Pain, Prevalence, Bristol Myers Squibb, Prednisone, Janssen Pharmaceuticals, Testosterone, PARP, Rucaparib, Urine, Neoplasm, Urethra, Johnson & Johnson, AstraZeneca, Docetaxel, STEAP1, Bone pain, AMG, Blood, ADT, LAS, Patient, Prostate-specific antigen, Astellas Pharma, HRR, Amgen, Prostate cancer, Swelling, Eli Lilly and Company, Hypoesthesia, Science, Growth, Merck & Co., Pressure, Pharmaceutical industry

LAS VEGAS, Dec. 20, 2023 /PRNewswire/ -- DelveInsight's Metastatic Castration-Resistant Prostate Cancer Market Insights report includes a comprehensive understanding of current treatment practices, mCRPC emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • As per DelveInsight analysis, the total metastatic castration-resistant prostate cancer market size in the 7MM was estimated to be nearly USD 6.4 billion in 2022, which is expected to show positive growth by 2032.
  • As per DelveInsight estimates, in 2022, total diagnosed prevalent cases of mCRPC were around 127K in the 7MM.
  • However, mCRPC remains a lethal diagnosis and more effective therapeutic approaches against mCRPC are necessary to improve clinical outcomes further.
  • The estimated five-year survival rate for men with metastatic prostate cancer is approximately 30%, in stark contrast to the 100% survival rate for those with localized prostate cancer.

Metastatic Castration-Resistant Prostate Cancer Market is Predicted to Exhibit Remarkable Growth at a CAGR of 7.5% During the Study Period (2019-2032), Estimates DelveInsight

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星期三, 十二月 20, 2023
GT0918, Niraparib, Phase, Ipsen, CRPC, Prognosis, Daiichi Sankyo, PSMA, BRCA, FDA, Spacecraft, Gesellschaft mit beschränkter Haftung, DNA, EU4, Cancer, Novartis, ICIS, Abiraterone acetate, Ageing, Urination, PSA, Metastasis, CYP17, Survival rate, Pain, Prevalence, Bristol Myers Squibb, Prednisone, Janssen Pharmaceuticals, Testosterone, PARP, Rucaparib, Urine, Neoplasm, Urethra, Johnson & Johnson, AstraZeneca, Docetaxel, STEAP1, Bone pain, AMG, Blood, ADT, LAS, Patient, Prostate-specific antigen, Astellas Pharma, HRR, Amgen, Prostate cancer, Swelling, Eli Lilly and Company, Hypoesthesia, Science, Growth, Merck & Co., Pressure, Pharmaceutical industry

LAS VEGAS, Dec. 20, 2023 /PRNewswire/ -- DelveInsight's Metastatic Castration-Resistant Prostate Cancer Market Insights report includes a comprehensive understanding of current treatment practices, mCRPC emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • As per DelveInsight analysis, the total metastatic castration-resistant prostate cancer market size in the 7MM was estimated to be nearly USD 6.4 billion in 2022, which is expected to show positive growth by 2032.
  • As per DelveInsight estimates, in 2022, total diagnosed prevalent cases of mCRPC were around 127K in the 7MM.
  • However, mCRPC remains a lethal diagnosis and more effective therapeutic approaches against mCRPC are necessary to improve clinical outcomes further.
  • The estimated five-year survival rate for men with metastatic prostate cancer is approximately 30%, in stark contrast to the 100% survival rate for those with localized prostate cancer.