Encephalopathy

CARVYKTI® is the First and Only BCMA-Targeted Treatment Approved by the U.S. FDA for Patients with Relapsed or Refractory Multiple Myeloma Who Have Received at Least One Prior Line of Therapy

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Saturday, April 6, 2024
Guillain–Barré syndrome, Bone marrow, Medical College of Wisconsin, Encephalopathy, Survival, Immunotherapy, BCMA, Patient, Hypotension, Mortality, B-cell maturation antigen, Edema, Periodic breathing, Associate professor, Dexamethasone, Certification, Neutropenia, Dizziness, Risk, Headache, Chills, Myelodysplastic syndrome, Coagulopathy, DPD, Johnson & Johnson, Immune system, Effect, Fatigue, Thrombocytopenia, Food, Disease, Drive, Appetite, Lymphocytopenia, JNJ, Constipation, Multiple myeloma, Acute myeloid leukemia, CRS, Death, Hypersensitivity, Cytopenia, Cytokine release syndrome, ASCO, Use, Hematology, FDA, PVD, Annual general meeting, T cell, DSM-IV codes, Incidence, Tachycardia, Bortezomib, Diarrhea, Viral, Hope, United, Cough, Daratumumab, Nausea, Society, Division, Physician, Pharmaceutical industry

HORSHAM, Pa., April 5, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) announced today that the U.S. Food and Drug Administration (FDA) has approved CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide.1 With this approval, CARVYKTI® becomes the first and only B-cell maturation antigen (BCMA)-targeted therapy approved for the treatment of patients with multiple myeloma as early as first relapse.

Key Points: 
  • "This milestone underscores our commitment to improve outcomes for patients and transform the treatment of multiple myeloma with CARVYKTI," said Jordan Schecter, M.D., Vice President, Disease Area Leader, Multiple Myeloma, Johnson & Johnson Innovative Medicine.
  • CARVYKTI® is a cell therapy that works by harnessing a patient's immune system, or T cells, to fight the disease.
  • Treatment requires extensive training, preparation, and certification to ensure a positive experience for patients.
  • Since initial approval in February 2022, Johnson & Johnson has made significant advances in manufacturing to rapidly scale CARVYKTI® production.

The United Mitochondrial Disease Foundation Announces Inaugural Venture Philanthropy Investment; The Mito Fund Invests $500k in Pierrepont Therapeutics, Inc.

Retrieved on: 
Thursday, April 4, 2024
Disease, Encephalopathy, Risk, Trustee, Patient, MNGIE, Research, Columbia University, Mitochondrial disease, Science, Death, MD, Malnutrition, Enzyme replacement therapy, UMDF, Natural history, Pharmaceutical industry

PITTSBURGH, April 4, 2024 /PRNewswire-PRWeb/ -- The United Mitochondrial Disease Foundation (UMDF) today announced the inaugural investment for its venture philanthropy initiative, The Mito Fund.

Key Points: 
  • PITTSBURGH, April 4, 2024 /PRNewswire-PRWeb/ -- The United Mitochondrial Disease Foundation (UMDF) today announced the inaugural investment for its venture philanthropy initiative, The Mito Fund.
  • "We've said all along that time is something mitochondrial disease families just don't have, especially when it comes to something as devastating as MNGIE.
  • Following an extensive review process, the Pierrepont investment was recommended by UMDF's Venture Philanthropy Investment Committee, an advisory group consisting of world-class experts in the areas of mitochondrial disease, pharmaceutical development, and venture investments.
  • UMDF thanks the Kallaos and Wright families for their founding gifts that helped make the launch of The Mito Fund possible.

Human medicines European public assessment report (EPAR): Kisplyx, lenvatinib, Date of authorisation: 25/08/2016, Revision: 21, Status: Authorised

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Friday, January 5, 2024
Marketing, Hypothyroidism, INN, Woman, Progression-free survival, Nausea, Syndrome, Heart, Cancer, Inflammation, Skull, Proteinuria, Anatomy, Cell division, IIA, Diarrhea, Fatigue, ATC, Capsule, Bleeding, Pancreas, RET, VEGF, FGFR, Cerebral edema, Neoplasm, Stroke, Enzyme, Liver failure, Safety, Kidney cancer, Arthralgia, Heart failure, Patient, Urine, Medical Subject Headings, Stomatitis, Immune system, Everolimus, International, Encephalopathy, Language, Somatic symptom disorder, Blood, Mouth, Catherine Disher, Select, Medicine, Vomiting, Pembrolizumab, Cholesterol, PDGF, Kidney failure, Weight loss, Headache, KIT, Brain, Acral lentiginous melanoma, Swelling, Hypoesthesia, Splenic infarction, Confusion, European Medicines Agency, RCC, Kidney, Growth, Sunitinib, Hypercholesterolemia, Rash, Medical device

Human medicines European public assessment report (EPAR): Kisplyx, lenvatinib, Date of authorisation: 25/08/2016, Revision: 21, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Kisplyx, lenvatinib, Date of authorisation: 25/08/2016, Revision: 21, Status: Authorised

Human medicines European public assessment report (EPAR): Yescarta, axicabtagene ciloleucel, Date of authorisation: 23/08/2018, Revision: 13, Status: Authorised

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Tuesday, January 2, 2024
Patient, Medical Subject Headings, Marketing, INN, Risk, Fever, Diffusion, DLBCL, ATC, Bone marrow, Pain, Cytokine release syndrome, International, Encephalopathy, Headache, Disease, Language, Blood, Hospital, Cancer, B-cell lymphoma, Myenteric plexus, Death, European Medicines Agency, Infection, PMBCL, Select, Cell, CD19, Time, Anatomy, Somnolence, Vomiting, IIA, Follicular lymphoma, Medical device

Human medicines European public assessment report (EPAR): Yescarta, axicabtagene ciloleucel, Date of authorisation: 23/08/2018, Revision: 13, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Yescarta, axicabtagene ciloleucel, Date of authorisation: 23/08/2018, Revision: 13, Status: Authorised

Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

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Tuesday, January 2, 2024
People, Marketing, INN, Risk, Leukopenia, Hypophosphatemia, Hypotension, Pneumonia, Potassium, Nausea, Cancer, Myenteric plexus, Infection, Anatomy, Cough, IIA, Multiple myeloma, Chills, Consciousness, Diarrhea, Fever, Fatigue, Bone marrow, ATC, Pharyngitis, Pain, Platelet, Disease, Nose, Edema, Neutrophil, Speech, Depression, Appetite, Writing, Lymphocyte, Safety, Water, Tachycardia, Anemia, Neurological disorder, Hypocalcemia, Sepsis, Hypokalemia, Patient, Medical Subject Headings, Febrile neutropenia, Lymphocytopenia, Cytokine release syndrome, International, Encephalopathy, Neutropenia, Blood, Language, Thrombocytopenia, Neurotoxicity, Select, Medicine, Vomiting, Upper respiratory tract infection, Headache, Hospital, BCMA, Confusion, European Medicines Agency, Cell, Medical device

Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Carvykti, ciltacabtagene autoleucel, Date of authorisation: 25/05/2022, Revision: 5, Status: Authorised

New Analyses Presented at ASH 2023 Support the Potential Long-Term Response and Safety of Kite’s Tecartus® in Patients With Aggressive Blood Cancers

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Tuesday, December 12, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Hospitals, Clinical Trials, Hackensack University Medical Center, Safety, Cytokine release syndrome, Senior, City, ASH, Aes, CRS, Haematopoietic system, Sepsis, Patient, Abstract, Thrombocytopenia, Blinatumomab, The James Cancer Hospital, Division, SCT, Acute lymphoblastic leukemia, Neutropenia, ORR, Encephalopathy, B-ALL, Infection, Survival, Death, CD20, Doctor of Philosophy, Society, Abstract (summary), OS, Confidence interval, TP53, LD, Cytopenia, Pharmaceutical industry, Nursing, Hematology, Brexucabtagene autoleucel, Lymphoma, MD

“The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.

Key Points: 
  • “The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.
  • The median OS for patients with complete response (CR) (n=46) was 58.7 months.
  • Efficacy and safety outcomes for 23 patients with R/R MCL enrolled in ZUMA-18, a multicenter, open-label, expanded-access study of Tecartus, were also presented.
  • An analysis of a CIBMTR observational database of R/R MCL patients receiving Tecartus from 84 U.S. centers was presented.

Entrada Therapeutics Reports Third Quarter 2023 Financial Results

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Tuesday, November 7, 2023
FDA, United Kingdom, Clinical trial, Vertex, Food, Dystrophin, Toxicology, Neurodegenerative disease, G&A, MNGIE, Research and development, CTA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, Volunteering, R, Enzyme replacement therapy, Pharmacokinetics, Male, Growth, Thymidine, Entrada, Duchenne muscular dystrophy, General Administration of Sport of China, Safety, DMD, Therapy, Patient, Administration, Encephalopathy, Pharmaceutical industry, IND, Duchenne

BOSTON, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV™)-therapeutics as a new class of medicines, today reported financial results for the third quarter ending September 30, 2023, and highlighted recent business updates.

Key Points: 
  • In July 2023, Entrada entered into a license agreement to advance the development of ENTR-501 with Pierrepont Therapeutics, Inc., a mitochondrial disease-focused company.
  • Research & Development (R&D) Expenses: R&D expenses were $22.2 million for the third quarter of 2023, compared to $19.0 million for the same period in 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $7.5 million for the third quarter of 2023, compared to $7.0 million for the same period in 2022.
  • Net Income (Loss): Net income was $35.5 million for the third quarter of 2023, compared to a net loss of $(25.1) million for the same period in 2022.

Praxis Precision Medicines Provides Portfolio Update at 2023 R&D Day

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Monday, October 2, 2023
Epilepsy, Seizure, Tremor, Central nervous system, University, Event, Week, Le bonheur, DSM-IV codes, NYU, Aes, New York University Grossman School of Medicine, Media, Part, CNS, Șaeș, Essential tremor, Professor, Patient, Master of Science, Webcast, FANA, Neurology, Encephalopathy, Propranolol, R, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, FAAN, Food, Medical imaging, Pharmaceutical industry, Drug, Nursing, Praxis

BOSTON, Oct. 02, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided an update on its clinical portfolio at the company’s R&D Day. Praxis’ leaders were joined by two esteemed key opinion leaders in movement disorders and epilepsy, respectively: Alberto J. Espay, M.D., MSc, FAAN, FANA, Professor and Endowed Chair of the James J. and Joan A. Gardner Center for Parkinson’s Disease at the University of Cincinnati, as well as Jacqueline French, M.D., Professor of Neurology at NYU Grossman School of Medicine and President, Director and Founder of the Epilepsy Study Consortium.

Key Points: 
  • Phase 3 (Essential3) program for ulixacaltamide initiating this quarter with mADL11 as primary endpoint; nearly 600 patients have already expressed interest in participating
    BOSTON, Oct. 02, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines , Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided an update on its clinical portfolio at the company’s R&D Day.
  • “We were thrilled to be able to share important updates from our portfolio.
  • Especially exciting is the early efficacy data we have seen with our PRAX-222 program in SCN2A gain-of-function developmental epilepsies and encephalopathies (DEEs).
  • A replay of the R&D day webcast will be available through the Events & Presentations page of the Investors + Media section of the company’s website at www.praxismedicines.com for the next 90 days.

Sentynl Therapeutics Announces Presentation of Study Evaluating the Efficacy and Safety of NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A at 2023 SSIEM Annual Symposium

Retrieved on: 
Thursday, September 7, 2023
Seizure, Consultant, Infection, European Medicines Agency, Encephalopathy, Survival, Necrotizing enterocolitis, Genetic distance, Cox, Whole genome sequencing, Biochemistry, Molecular medicine, Ministry, Fosdenopterin, RSV, Patient, University of Cologne, Risk, Chemistry, Growth, Mortality, Saint Mary's Hospital, Hospital, Rare, Caregiver, Therapy, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Molybdenum cofactor deficiency, Pneumonia, Food, Safety, Pharmaceutical industry, Metabolism, Health

SOLANA BEACH, Calif., Sept. 7, 2023 /PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases, announced that data evaluating NULIBRY® (fosdenopterin) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A were presented at the 2023 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Meeting on Aug. 30 in Jerusalem. NULIBRY is indicated to reduce the risk of mortality in patients with MoCD Type A, an ultra-rare, autosomal recessive, inborn error of metabolism causing sulfite-induced neurodegeneration and early death.

Key Points: 
  • NULIBRY is indicated to reduce the risk of mortality in patients with MoCD Type A, an ultra-rare, autosomal recessive, inborn error of metabolism causing sulfite-induced neurodegeneration and early death.
  • "Early treatment with fosdenopterin (NULIBRY) improves survival and neurodevelopmental outcomes, reducing burden on patients and caregivers."
  • Treatment with rcPMP/NULIBRY led to a normalization of MoCD Type A-associated urinary biomarker levels.
  • To further evaluate the safety and efficacy of NULIBRY, a post-approval non-interventional study is being initiated in Europe.

The International Lyme and Associated Diseases Society Brings Annual Scientific Conference to Boston

Retrieved on: 
Thursday, June 29, 2023
Impact, International Lyme and Associated Diseases Society, Population biology, Practice management software, Pulse, Science, MD, Patient, Tick-borne disease, Encephalopathy, Conference, Content, ILADS, Nursing, Pediatrics

CHEVY CHASE, Md., June 29, 2023 /PRNewswire/ -- The International Lyme and Associated Diseases Society (ILADS) and its foundation, the International Lyme and Associated Diseases Educational Foundation (ILADEF), will welcome its 24th Annual Scientific Conference to Boston, MA.

Key Points: 
  • Highlights Include Expanded Learning Tracks, Enhanced Networking Time, & Refreshed Approach to Content
    CHEVY CHASE, Md., June 29, 2023 /PRNewswire/ -- The International Lyme and Associated Diseases Society (ILADS) and its foundation, the International Lyme and Associated Diseases Educational Foundation (ILADEF), will welcome its 24 th Annual Scientific Conference to Boston, MA.
  • ILADS Scientific Conference is the premier educational experience for healthcare professionals caring for patients with tick-borne disease.
  • "This year's theme is Vector-Borne Diseases: Understanding the Impact of Microbial Communities," says Sarah Quillen, ILADS Executive Director.
  • If you are a member of the media who would like to obtain a press pass, email [email protected] .