Bone pain

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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Wednesday, April 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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Tuesday, April 9, 2024
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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

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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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Tuesday, April 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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Monday, April 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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Tuesday, January 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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Tuesday, January 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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Tuesday, January 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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Wednesday, December 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

Retrieved on: 
Wednesday, December 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.

GPCR Therapeutics Announces Publication on Improving Hematopoietic Stem Cell Mobilization Using Propranolol with GPC-100

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Thursday, October 26, 2023
Stem cell, Survival, Bone pain, Gq alpha subunit, Google Store, GPCR, Multiple myeloma, Propranolol, Therapy, Hope, Health, Hematological Cancer Research Investment and Education Act, CXCR4, Sickle cell disease, Research, Cell, ASCT, Mouse, Clinical trial, Risk, Cancer, HSC, G-CSF, G protein-coupled receptor, Transplant, Bone marrow, PLOS, Tumors of the hematopoietic and lymphoid tissues, Biology, Patient, B cell, Seoul National University, Pharmaceutical industry, Microbiology

These findings strongly support the use of this combination for peripheral blood HSC harvest in autologous stem cell transplant treatment in hematological cancers.

Key Points: 
  • These findings strongly support the use of this combination for peripheral blood HSC harvest in autologous stem cell transplant treatment in hematological cancers.
  • The paper, authored in collaboration with Seoul National University’s School of Biological Sciences and Institute of Microbiology, is entitled, “GPC-100, a novel CXCR4 antagonist, improves in vivo hematopoietic cell mobilization when combined with propranolol” (1).
  • The authors highlight that GPC-100 alone shows greater mobilization efficacy than a drug already approved for stem cell mobilization.
  • Increasingly, clinicians are using Autologous Stem Cell Transplant (ASCT) to treat patients, which has improved the anti-cancer response and survival compared to conventional chemotherapy.