Adrenal insufficiency

Corcept Announces Positive Results From Open-Label Portion of Pivotal Phase 3 GRACE Trial in Patients With Cushing’s Syndrome

Retrieved on: 
Monday, April 22, 2024
GRACE, Hyperglycemia, University, Patient, Syndrome, SBP, Adrenal insufficiency, Safety, NDA, Waist, Ann Arbor, Michigan, MD, Doctor of Philosophy, Vaginal bleeding, DSM-IV codes, Doctor of Pharmacy, Diabetes, Hypertension, Metabolism, Internal medicine, Endocrinology, Division, Deficit spending, DBP, Pharmaceutical industry

In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.

Key Points: 
  • In the “open-label” phase, 152 patients with Cushing’s syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks.
  • GRACE’s primary endpoint is maintenance of blood pressure control in the “randomized withdrawal” phase, with maintenance of glycemic control as the key secondary endpoint.
  • Other key secondary and exploratory endpoints in the randomized withdrawal phase include changes in weight, waist circumference, cognitive impairment and Cushing’s Quality of Life score.
  • Patients in the open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia and other key secondary and exploratory endpoints.

Neurocrine Biosciences Launches WHAT THE C@H?! Educational Initiative to Support Congenital Adrenal Hyperplasia Community

Retrieved on: 
Wednesday, April 24, 2024
Glucocorticoid, Neurocrine Biosciences, CAH, Adrenal insufficiency, Neurocrine, Research, Parent, Life, Congenital adrenal hyperplasia, CRF1, Patient, Caregiver, SAN, Pharmaceutical industry

provides a platform for the congenital adrenal hyperplasia (CAH) community to find educational information, share experiences and learn about current research.

Key Points: 
  • provides a platform for the congenital adrenal hyperplasia (CAH) community to find educational information, share experiences and learn about current research.
  • SAN DIEGO, April 24, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the launch of WHAT THE C@H?!
  • , an educational initiative that aims to close the gap in the need for helpful information about congenital adrenal hyperplasia (CAH) and acknowledges the frustrations and challenges experienced by the community in managing the condition.
  • "We hope this educational initiative, which was informed by insights from the community, is helpful to those navigating and managing this difficult condition."

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Monday, April 8, 2024
Diabetes, Anaphylaxis, IgA nephropathy, Glaucoma, CYP3A4, Dermatitis, Patient, Parasitism, Indinavir, Budesonide, Ritonavir, Quality of life, Peripheral edema, UPCR, Drug interaction, Vaccine, Saquinavir, Erythromycin, Congress, White, Risk, Headache, Spasm, Ketoconazole, Immunoglobulin A, Liver, Adrenal insufficiency, Immune system, Prediabetes, Proteinuria, Hypertension, Contraindication, Arthralgia, Ciclosporin, International Society, Immunosuppression, KOL, Acne, Infection, EGFR, Measles, Miscarriage, ISN, Hypersensitivity, Pregnancy, HPA, Chickenpox, Fetus, Birth, Itraconazole, CALT, Upper respiratory tract infection, Osteoporosis, Kidney, Cataract, Tropical ulcer, Pharmaceutical industry

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

Retrieved on: 
Monday, April 8, 2024
Diabetes, Anaphylaxis, IgA nephropathy, Glaucoma, CYP3A4, Dermatitis, Patient, Parasitism, Indinavir, Budesonide, Ritonavir, Quality of life, Peripheral edema, UPCR, Drug interaction, Vaccine, Saquinavir, Erythromycin, Congress, White, Risk, Headache, Spasm, Ketoconazole, Immunoglobulin A, Liver, Adrenal insufficiency, Immune system, Prediabetes, Proteinuria, Hypertension, Contraindication, Arthralgia, Ciclosporin, International Society, Immunosuppression, KOL, Acne, Infection, EGFR, Measles, Miscarriage, ISN, Hypersensitivity, Pregnancy, HPA, Chickenpox, Fetus, Birth, Itraconazole, CALT, Upper respiratory tract infection, Osteoporosis, Kidney, Cataract, Tropical ulcer, Pharmaceutical industry

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

OrthoPediatrics Corp. Launches New RESPONSE Rib and Pelvic Fixation System for EOS Scoliosis

Retrieved on: 
Monday, March 4, 2024
EOS, Rib, Elective surgery, Orthopediatrics, Adrenal insufficiency, Surgeon, Family, Scoliosis, Patient, Risk, Dentistry

WARSAW, Ind., March 04, 2024 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS a company focused exclusively on advancing the field of pediatric orthopedics, today announced it has launched a new RESPONSE™ Rib and Pelvic Fixation system to treat children with Early Onset Scoliosis (“EOS”).

Key Points: 
  • WARSAW, Ind., March 04, 2024 (GLOBE NEWSWIRE) -- OrthoPediatrics Corp. (“OrthoPediatrics” or the “Company”) (Nasdaq: KIDS a company focused exclusively on advancing the field of pediatric orthopedics, today announced it has launched a new RESPONSE™ Rib and Pelvic Fixation system to treat children with Early Onset Scoliosis (“EOS”).
  • The new addition to the RESPONSE™ family includes implants and instruments for rib and pelvic fixation, and associated devices to connect the fixation points.
  • As an adjunct to the foundational RESPONSE™ Scoliosis System, the Rib and Pelvic system is compatible with 5.5/6.0 and 4.5/5.0 RESPONSE™ Systems, allowing surgeons to treat the entire spectrum of patients with scoliosis.
  • Greg Odle, President of the OrthoPediatrics’ Scoliosis Division echoed his excitement for the launch of this new system, saying “We are excited to enter the Early Onset Scoliosis treatment market with the RESPONSE™ Rib & Pelvic System.

The Wyss Foundation Provides $5 Million in Funding for Children's Hospital of Philadelphia's Wyss/Campbell Center for Thoracic Insufficiency Syndrome

Retrieved on: 
Thursday, February 22, 2024
VEPTR, Division, Adrenal insufficiency, Algorithm, Spinal fusion, Spine, Education, MD, Children's Hospital of Philadelphia, Physician, FDA, Quality of life, 3D, Hospital, Surgeon, Health, Wyss, TIS, Patient, CHOP, Child, Wyss Foundation, Nursing

PHILADELPHIA, Feb. 22, 2024 /PRNewswire/ -- Children's Hospital of Philadelphia (CHOP) is thrilled to announce a gift of $5 million from the Wyss Foundation to support innovations that improve the lives of patients with thoracic insufficiency syndrome (TIS). An initial gift from Hansjörg Wyss, philanthropist and chairman of the Wyss Foundation, was made to CHOP in 2019 and honored the legacy of the late Dr. Robert Campbell, a world-renowned CHOP pediatric orthopaedic surgeon. Now, additional funding has renamed and established the Wyss/Campbell Center for Thoracic Insufficiency Syndrome, one of CHOP's frontier programs, a trailblazing group of initiatives pioneering new advances in children's health. 

Key Points: 
  • Now, additional funding has renamed and established the Wyss/Campbell Center for Thoracic Insufficiency Syndrome , one of CHOP's frontier programs, a trailblazing group of initiatives pioneering new advances in children's health.
  • The $5 million gift will ensure the Center, its clinical team, researchers and patients have the resources needed to thrive by amplifying studies, tools and education.
  • The Center will be named Wyss/Campbell Center for Thoracic Insufficiency Syndrome in perpetuity.
  • The Wyss Foundation is honored to support the team working to improve the quality of life for children living with TIS," said Molly McUsic, President of the Wyss Foundation.

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Sunday, February 4, 2024
Hearing, Inflammation, Civil service commission, Knowledge, European, Cell, Haematopoiesis, COMP, IRIS, Bone marrow, Mitochondrion, Adrenal insufficiency, Regulation, Gland, ABCD1, Patient, Safety, Diagnosis, EMA, ALD, European Commission, Adrenoleukodystrophy, Seizure, B cell, PPAR, Brain, Disease, Therapy, Eurostat, Tissue, Dementia, Medicine, Committee, Hormone, DSM-IV codes, Spinal cord, Pharmaceutical industry

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Additional ODM-208/MK-5684 Phase II data presented at ASCO-GU 2024

Retrieved on: 
Friday, January 26, 2024
Senior, Hormone replacement therapy, Patient, Prostate cancer, ASCO, Prostate-specific antigen, CYP11A1, LBD, Mutation, Adrenal insufficiency, PSA, Safety, Research, Cancer, Growth, Pharmaceutical industry

Orion presented yesterday at the 2024 ASCO GU Cancers Symposium a poster with additional data from the ongoing Phase II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC) with and without AR-LBD mutations.

Key Points: 
  • Orion presented yesterday at the 2024 ASCO GU Cancers Symposium a poster with additional data from the ongoing Phase II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC) with and without AR-LBD mutations.
  • The new data reports initial results in an extension cohort of mainly AR-LBD wild-type patients combined with previously reported phase 2 data (AR-LBD mutation-positive only).
  • Data from the Phase II CYPIDES trial suggest that ODM-208/MK-5684 potently inhibits all steroid-hormone biosynthesis with observed antitumor activity in a heavily pretreated mCRPC population, especially in patients with AR-LBD mutations.
  • In Phase II CYPIDES, at data cut-off, a total of 134 previously treated mCRPC patients received 5mg of ODM-208/MK-5684 twice a day with glucocorticoid/mineralocorticoid replacement and ongoing androgen-deprivation therapy.

NEJM Evidence publishes results from Phase I/IIa CYPIDES trial with ODM-208

Retrieved on: 
Wednesday, December 27, 2023
Prostate cancer, Adrenal insufficiency, Orion, LBD, Hormone replacement therapy, NEJM, Research, Testosterone, Mutation, Prostate-specific antigen, Patient, Safety, CYP11A1, Senior, Pharmaceutical industry

The NEJM Evidence has published interim data from the first part of Phase I/II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC).

Key Points: 
  • The NEJM Evidence has published interim data from the first part of Phase I/II CYPIDES trial evaluating the safety and efficacy of ODM-208 (or MK-5684), an investigational, oral CYP11A1 inhibitor, in heavily pretreated patients with metastatic castration-resistant prostate cancer (mCRPC).
  • Results from the Phase I/IIa CYPIDES trial suggest that ODM-208 potently inhibits all steroid-hormone biosynthesis with observed antitumor activity in a heavily pretreated mCRPC population, especially in patients with androgen receptor gene (AR) ligand-binding-domain (LBD) mutations.
  • In Phase I/IIa CYPIDES, a total of 92 previously treated mCRPC patients received 5mg of ODM-208 twice a day with glucocorticoid/mineralocorticoid replacement and ongoing androgen-deprivation therapy.
  • In phase I, 20 out of the total 47 patients had AR LBD mutation.

Food And Drug Administration Grants Two New Label Changes To Tirosint®-Sol (Levothyroxine Sodium) Oral Solution

Retrieved on: 
Monday, December 4, 2023
Biotechnology, FDA, Managed Care, Health, General Health, Women, Seniors, Pharmaceutical, Research, Consumer, Science, Breakfast, Heat intolerance, Absorption, Food, Hypothyroidism, Eating, Commercial Operating System, Irritability, Headache, Risk, Tremor, Arrhythmia, IBSA, Rash, Cardiovascular disease, Insurance, Patient, Diabetes, Myocardial infarction, Periodic breathing, Appetite, Atrial fibrillation, Muscle weakness, Enzyme inhibitor, Adrenal insufficiency, Diarrhea, Menstruation, University, Water, Insomnia, PPI, Glycerol, Common, Spasm, Nervous, Hyperthyroidism, Weight loss, Pharmacy, Pharmaceutical industry, Dietary supplement, Levothyroxine

The U.S. Food and Drug Administration (FDA) has approved two new changes to the label of Tirosint-SOL (levothyroxine sodium) oral solution, a unique formulation of levothyroxine (LT4) for the treatment of hypothyroidism.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) has approved two new changes to the label of Tirosint-SOL (levothyroxine sodium) oral solution, a unique formulation of levothyroxine (LT4) for the treatment of hypothyroidism.
  • The first regards the use of Tirosint-SOL in the presence of proton pump inhibitor (PPI) therapy.
  • Both label changes help to differentiate Tirosint-SOL from other levothyroxine therapies.
  • Similarly, current labeling for all levothyroxine sodium therapies instructs patients to self-administer the drug once daily, on an empty stomach, one-half to one hour before breakfast.