Lymphadenopathy

New tiopronin delayed-release tablets launch with patient support for the treatment of cystinuria in the US

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Lundi, mai 13, 2024
FDA, Health, Research, Pharmaceutical, Science, Biotechnology, Partnership, Incidence, Patient, Rash, Lymphadenopathy, Full, Diarrhea, Birth, Milk, Label, Generic, Risk, Tiopronin, Food, Pregnancy, Education, Nausea, Glomerulonephritis, Fever, Arthralgia, SVP, Vomiting, Tablet, Cystinuria, Torrent, Kidney stone disease, Kidney, Breastfeeding, Miscarriage, Proteinuria, Fatigue, Hyperthermia, Child, Lactation, Cycle, Use, Nephrotic syndrome, Birth control

In addition, a Patient Nutrition Program* will be available to reinforce the clinic’s nutrition plan,” says Chikai Lai, SVP & Chief Commercial Officer, Cycle.

Key Points: 
  • In addition, a Patient Nutrition Program* will be available to reinforce the clinic’s nutrition plan,” says Chikai Lai, SVP & Chief Commercial Officer, Cycle.
  • Alcohol: Tiopronin is released faster from tiopronin delayed-release tablets in the presence of alcohol and the risk for adverse events associated with tiopronin delayed-release tablets when taken with alcohol is unknown.
  • Because of the potential for serious adverse reactions, including nephrotic syndrome, advise patients that breastfeeding is not recommended during treatment with tiopronin delayed-release tablets.
  • Tiopronin delayed-release tablets are not approved for use in pediatric patients weighing less than 20 kg or in pediatric patients unable to swallow tablets.

Praxis Medical Announces FDA Clearance of the EndoCore EBUS-TBNA Biopsy Device

Retrieved on: 
Mardi, octobre 17, 2023
Oncology, Health, FDA, Medical Devices, Hospitals, Surgery, Health Technology, Diagnosis, Lung cancer, Food, EBUS, Standard of care, Lymphoma, Patient, Lymphadenopathy, Biopsy, Sarcoidosis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Metastasis, Neoplasm, Morton Plant Hospital, Malignancy, Physician, Medical imaging

Praxis Medical announced that the U.S. Food and Drug Administration (FDA) has granted 510(k) clearance of the EndoCore EBUS-TBNA fine needle biopsy device.

Key Points: 
  • Praxis Medical announced that the U.S. Food and Drug Administration (FDA) has granted 510(k) clearance of the EndoCore EBUS-TBNA fine needle biopsy device.
  • This procedure is performed by inserting a long, flexible fine needle through a bronchoscope for biopsy and pathological analysis.
  • The device is powered by a motor that rotates the needle during biopsy to facilitate cellular harvesting.
  • “We expect the EndoCore device will collect larger samples of tissue with higher cellularity which could translate to higher success rates for ancillary testing.

DRESS Smarter: Bridging the Gap in Severe Drug Reactions

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Mercredi, juillet 12, 2023
Knowledge, Hypersensitivity, Eosinophilia, Ohio State University, Disulfiram-like drug, Vancomycin, Heart, Anticonvulsant, Education, World Down Syndrome Day, Smarter Sentencing Act, Fever, Trimethoprim/sulfamethoxazole, Human, Research, Drug reaction with eosinophilia and systemic symptoms, Allopurinol, Rash, Plasma protein binding, DRESS, Patient, Physician, Risk, Ohio State University Wexner Medical Center, REDRESS, Liver, Diagnosis, Health, Recurrence, Lymphadenopathy, Residential care, Pharmaceutical industry, Nursing, Medical device, Vaccine, Lamotrigine

RICHMOND, Va., July 12, 2023 /PRNewswire-PRWeb/ -- Around the world, millions of people suffer from severe adverse drug reactions, many of which do not get proper funding or research. For patients with DRESS Syndrome, the experience can be isolating. However, a nonprofit behind National DRESS Syndrome Day is providing a platform for building awareness of this debilitating life threatening condition. On July 16, 2023, the DRESS Syndrome Foundation will encourage patients and medical communities to unite over this year's theme: "DRESS/smarter." By recognizing positive advancements in DRESS Syndrome, the holiday gives a voice to this uncommon but life-threatening reaction, and supports research efforts that bridge the gap worldwide.

Key Points: 
  • A national holiday with a global reach, DRESS Syndrome Day highlights progress and future insights into severe adverse drug reactions.
  • "There is still so much we don't understand about this severe and potentially fatal drug reaction," explains Tasha Tolliver, Executive Director and Co-Founder of DRESS Syndrome Foundation.
  • Additionally, Dr. Korman's team identified a unique "earlobe crease sign" that distinguishes DRESS from other severe adverse drug reactions, facilitating faster and more accurate diagnosis.
  • "We founded the DRESS Syndrome Foundation to fill a much-needed gap in patient advocacy, a gap that today would have saved our daughters lives," says Nancy Szakacsy, Co-Founder of DRESS Syndrome Foundation.

AVROBIO Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Update

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Jeudi, mars 23, 2023
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, GMP, Tissue, VCN, University of California, San Diego Performance-Based Skills Assessment, Gaucher's disease, Enteropathy, University, Safety, Health care, Food, CMC, Aes, Clinical trial, MPS II, Skin, Glycogen storage disease, Cerebrospinal fluid, MPS, CNS, IDS, Cystinosis, AVRO, ERT, HSPC, Patient, Physician, Risk, Medicines and Healthcare products Regulatory Agency, Liver, CSF, HSC, UOM, FDA, Syndrome, Enzyme replacement therapy, Lymphadenopathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hunter syndrome, GI, Cystine, DSM-IV codes, MHRA, Disease, Pharmaceutical industry, Vaccine, Gaucher

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.
  • “2022 was a transformative year for AVROBIO, topped in December with a robust clinical and regulatory update for our Gaucher disease program.
  • Safety data to date from GD1 and GD3 patients indicate no adverse events (AEs) related to drug product.
  • No major chemistry, manufacturing and controls (CMC) changes are anticipated for AVROBIO’s plato® gene therapy platform as the company prepares to enter a registrational trial for GD3.

EISAI TO PRESENT LATEST DATA ON FYCOMPA® (PERAMPANEL) CIII AT THE 76TH AMERICAN EPILEPSY SOCIETY (AES) ANNUAL MEETING

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Mercredi, novembre 23, 2022
Suicidal ideation, Neglected tropical diseases, Marketing, Clinical trial, Epilepsy, Headache, Hospital, Anticonvulsant, AED, Risk, Plasma, Behavior, Anxiety, Seizure, Weight gain, Fever, Bruise, Death, Anger, Disease, DRESS, Vomiting, Depression, Mortality, Gait, Vertigo, Falls, Health, Somnolence, Patient, Pharmacotherapy, Ageing, Lymphadenopathy, Irritability, Dizziness, Ataxia, Head Injuries, Caregiver, Aggression, Human, Rash, Psychiatry, Self-harm, Central nervous system, United Nations Sustainable Development Group, AMPA, Fatigue, Fracture, Perampanel, Sustainable Development Goals, CNS, Homicidal ideation, Neurology, Nausea, Abdominal pain, POS, SUDEP, CIII, Personality, Pharmaceutical industry, Residential care, Eisai

NUTLEY, N.J., Nov. 23, 2022 /PRNewswire/ -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") announced today that the company will have a total of 34 posters, including the latest data on its in-house discovered and developed anti-seizure medication (ASM) FYCOMPA® (perampanel) CIII, at the 76th American Epilepsy Society Annual Meeting (AES 2022), to be held in Nashville, Tennessee and virtually from December 2-6, 2022.

Key Points: 
  • Epilepsy is one of the most common neurological disorders, which affects 3.4 million people in the United States, including 470,000 children.
  • Partial-onset seizures are the most common types of seizures seen in people with epilepsy, accounting for 60 percent of all seizures.
  • Missed medication doses are the number one cause of breakthrough seizures, which can cause significant injury to patients.
  • Physicians are advised to recommend that pregnant patients taking FYCOMPA enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry.

Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib

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Mercredi, septembre 28, 2022
Attention deficit hyperactivity disorder predominantly inattentive, MPH, PDK1, B cell, Time, Safety, Lymphadenopathy, COVID-19, Autoimmunity, Risk, FDA, Food, Neutrophil, Phase, U.S. Securities and Exchange Commission, Gene, Population, Company, Degenerative disease, Macrophage, NDA, PDUFA, Lists of diseases, Metabolism, PIK3R1, Patient, PI3K, AKT, Cell, Euronext Amsterdam, Genetic testing, Annual report, Primary immunodeficiency, PIP3, Priority review, New Drug Application, APDS, NASDAQ, US Foods, Growth hacking, Index, Classification, Lymphoma, PIK3CD, Disease, Disclosure, Immunodeficiency, Pharmaceutical industry, Vaccine, MD, Pharming

LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, aligned with a Priority Review classification.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA)goaldate of March 29, 2023, aligned with a Priority Review classification.
  • Furthermore, safety data from the study showed that leniolisib was well tolerated by participants.
  • Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "The FDA's acceptance for priority review of Pharming's New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases.

Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib

Retrieved on: 
Mercredi, septembre 28, 2022
Attention deficit hyperactivity disorder predominantly inattentive, MPH, PDK1, B cell, Time, Safety, Lymphadenopathy, COVID-19, Autoimmunity, Risk, FDA, Food, Neutrophil, Phase, U.S. Securities and Exchange Commission, Gene, Population, Company, Degenerative disease, Macrophage, NDA, PDUFA, Lists of diseases, Metabolism, PIK3R1, Patient, PI3K, AKT, Cell, Euronext Amsterdam, Genetic testing, Annual report, Primary immunodeficiency, PIP3, Priority review, New Drug Application, APDS, NASDAQ, US Foods, Growth hacking, Index, Classification, Lymphoma, PIK3CD, Disease, Disclosure, Immunodeficiency, Pharmaceutical industry, Vaccine, MD, Pharming

LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, aligned with a Priority Review classification.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA)goaldate of March 29, 2023, aligned with a Priority Review classification.
  • Furthermore, safety data from the study showed that leniolisib was well tolerated by participants.
  • Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "The FDA's acceptance for priority review of Pharming's New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases.

Pfizer-BioNTech COVID-19 Vaccine Demonstrates Strong Immune Response, High Efficacy and Favorable Safety in Children 6 Months to Under 5 Years of Age Following Third Dose

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Lundi, mai 23, 2022
RNA transfection, NEW, Food, COVID 19, Pericarditis, Omicron, Allergy, EMA, Headache, Face, Vaccine, Center, Lymph, Nausea, Arthralgia, Severe acute respiratory syndrome coronavirus 2, COVID-19, Vaccination, United Kingdom, BioNTech, Vomiting, CDC, Syncope, Diarrhea, NYSE, Inflammation, Rash, Fever, Dizziness, US Foods, Seek, EUA, Emergency Use Authorization, Association, FDA, Appetite, VAERS, GMT, Myalgia, Heart, Fatigue, Lymphadenopathy, PFE, COVID, Pfizer–BioNTech COVID-19 vaccine, Population, Regulation of tobacco by the U.S. Food and Drug Administration, Chills, Coronavirus, Nasdaq, Hope, Safety, Union, BNTX, Korea Disease Control and Prevention Agency, FD, CEO, Pharmaceutical industry, Pfizer

Following a third dose in this age group, the vaccine was found to elicit a strong immune response, with a favorable safety profile similar to placebo.

Key Points: 
  • Following a third dose in this age group, the vaccine was found to elicit a strong immune response, with a favorable safety profile similar to placebo.
  • Vaccine efficacy, a secondary endpoint in this trial, was 80.3% in children 6 months to under 5 years of age.
  • Three 3-g doses of the Pfizer-BioNTech COVID-19 Vaccine was well-tolerated in this age group, and no new safety signals were identified.
  • The Pfizer-BioNTech COVID-19 Vaccine authorized for use in children 5 through 11 years of age should not be used interchangeably with COMIRNATY (COVID-19 Vaccine, mRNA).

Cellectar Biosciences Announces Positive DMC Review of Pivotal Trial of Iopofosine in Waldenstrom’s Macroglobulinemia

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Mardi, avril 26, 2022
Patient, Rituximab, News, ASCO, Lymphoma, Christian democracy, Clinical trial, Marketing, DMC, Brain, U.S. Securities and Exchange Commission, 131, COVID-19, Twitter, Security (finance), CLR, NASDAQ, Ibrutinib, Research, NHL, Haematopoietic system, Drug discovery, Regulation, Incidence, PDC, Salvage therapy, Lymphatic system, Antibody, Lymph, GLOBE, B cell, Multiple myeloma, Tissue, LinkedIn, Cytopenia, End organ damage, Review, Waldenström's macroglobulinemia, Language, Neuroblastoma, Bone marrow, Exchange, LPL, Lymphadenopathy, Immunoglobulin M, Safety, Data monitoring committee, FDA, Prevalence, Disease, Risk, Cancer, Sarcoma, Pharmaceutical industry, Vaccine, Facebook, WM

The pivotal trial is a global, non-comparator, single-arm, open-label expansion cohort of the currently ongoing Phase 2 CLOVER-1 study of CLR 131.

Key Points: 
  • The pivotal trial is a global, non-comparator, single-arm, open-label expansion cohort of the currently ongoing Phase 2 CLOVER-1 study of CLR 131.
  • The DMC is an independent committee of clinical research experts who review data in ongoing clinical trials.
  • The DMC assessment was based on a pre-specified futility analysis within the first 10 patients as defined in the study protocol.
  • Waldenstroms macroglobulinemia (WM) is a rare and incurable disease defined by specific genotypic subtypes that defines patient responses and long-term outcomes.

Pfizer and BioNTech Announce Data Demonstrating High Immune Response Following a Booster Dose of Their COVID-19 Vaccine in Children 5 Through 11 Years of Age

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Jeudi, avril 14, 2022
EMA, COVID-19, Seek, Blood, 2020 European Medicines Agency cyberattack, Dizziness, EUA, BNTX, FDA, Diarrhea, Allergy, US Foods, Food, Chills, Fever, Pericarditis, Serum, Severe acute respiratory syndrome coronavirus 2, Coronavirus, Nausea, Pfizer–BioNTech COVID-19 vaccine, Profile, Appetite, CDC, COVID 19, European Medicines Agency, Lymph, Vaccination, Heart, Nasdaq, United Kingdom, Food and Drug Administration, BioNTech, GM GMT platform, PFE, Safety, VAERS, RNA transfection, Vomiting, Association, Myalgia, Center, Korea Disease Control and Prevention Agency, FD, Vaccine, Antiserum, Inflammation, Headache, Fatigue, NEW, NYSE, Lymphadenopathy, Union, Face, Omicron, GMT, Emergency Use Authorization, COVID, Arthralgia, Pharmaceutical industry, Pfizer

These data demonstrate an increase in SARS-CoV-2 Omicron variant and wild-type strain neutralizing titers following a booster dose of the Pfizer-BioNTech COVID-19 vaccine compared to two doses.

Key Points: 
  • These data demonstrate an increase in SARS-CoV-2 Omicron variant and wild-type strain neutralizing titers following a booster dose of the Pfizer-BioNTech COVID-19 vaccine compared to two doses.
  • Pfizer and BioNTech plan to submit a request for Emergency Use Authorization (EUA) of a booster dose for children ages 5 through 11 in the U.S. in the coming days.
  • The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTechs proprietary mRNA technology, was developed by both BioNTech and Pfizer.
  • The Pfizer-BioNTech COVID-19 Vaccine authorized for use in children 5 through 11 years of age should not be used interchangeably with COMIRNATY (COVID-19 Vaccine, mRNA).