Calvarial doughnut lesions-bone fragility syndrome

Defeat Duchenne Canada and Parent Project Muscular Dystrophy Award $300,000 (USD) Clinical Fellowship in Duchenne Endocrinology and Bone Fragility

Retrieved on: 
Friday, November 3, 2023
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WASHINGTON and OTTAWA, ON, Nov. 3, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), and Defeat Duchenne Canada, Canada's leading charity fighting to defeat Duchenne, today announced a collaborative research award of $300,000 (USD) in support of two Clinical Fellowships in Duchenne Endocrinology and Bone Fragility. The three-year award will sponsor the extended fellowship of Dr. Kim Phung, as well as new fellow Dr. Rana Halloun, under the guidance of Dr. Leanne Ward, Professor of Pediatrics and Research Chair in Pediatric Bone Disorders at the University of Ottawa.

Key Points: 
  • Duchenne is the most common muscular dystrophy in children, affecting approximately one in 5,000 live male births.
  • Duchenne is caused by a change in the dystrophin gene that leads to progressive muscle weakness.
  • In 2021, Dr. Phung was awarded a two-year fellowship in pediatric endocrine and bone diseases at the University of Ottawa, with specific focus on endocrine and bone complications of Duchenne.
  • Ward and Phung will delve into the latest developments and crucial topics related to bone health, growth, puberty, and adrenal insufficiency for Duchenne muscular dystrophy.

PPMD Applauds FDA for Approval of AGAMREE® (Vamorolone) for Duchenne Patients Ages Two Years and Older

Retrieved on: 
Friday, October 27, 2023
Knowledge, FDA, Policy, Food, Arkansas Children's Hospital, Catalyst Pharmaceuticals, Research, Vamorolone, PPMD, Becker muscular dystrophy, Duchenne muscular dystrophy, Calvarial doughnut lesions-bone fragility syndrome, Insulin resistance, Patient, Hospital, Food and Drug Administration Amendments Act of 2007, Pharmaceutical industry, Becker, Duchenne

AGAMREE® marks a significant milestone as the second therapy approved for Duchenne that is applicable regardless of the specific genetic mutation.

Key Points: 
  • AGAMREE® marks a significant milestone as the second therapy approved for Duchenne that is applicable regardless of the specific genetic mutation.
  • Today's approval has the potential to offer patients a therapy option with better tolerability, addressing a significant unmet need in our community," said PPMD Founding President and CEO Pat Furlong.
  • We also want to commend FDA leadership for recognizing the potential of AGAMREE® to make a difference in the lives of individuals living with Duchenne," Furlong added.
  • "PPMD is pleased to have collaborated with other foundations and community partners to support the early development of vamorolone.

Preclinical Data Demonstrate Anti-Siglec-15 Treatment Reduced Bone Loss and Enhanced Bone Quality in Mice with Moderate-to-Severe Osteogenesis Imperfecta (OI)

Retrieved on: 
Tuesday, October 17, 2023
Hospital for Special Surgery, Male, Cortex, Spectroscopy, Female, BMD, Bone, Bone disease, Osteogenesis imperfecta, Society, Mab, Hospital, American Society for Bone and Mineral Research, Osteoporosis, Cancer, Fracture, Osteoclast, Calvarial doughnut lesions-bone fragility syndrome, Mouse, S15, Porosity, Life, OI, Patient, Medical imaging, Birth control

These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.

Key Points: 
  • These results support development of NC605 as a potential highly effective treatment for osteogenesis imperfecta, a rare disease in which bones easily fracture.
  • Osteogenesis imperfecta is a rare disorder that results in high bone turnover, abnormal bone formation, bone fragility and recurrent fractures.
  • There is no cure for OI and current anti-resorptive treatments increase bone mineral density (BMD) primarily by inhibiting bone loss; however, these agents also inhibit bone formation.
  • Unlike anti-resorptive therapies, NC605 enhances osteoblast recruitment, resulting in overall enhanced bone quality.

Medimaps Group Receives EU Certification Under the Medical Device Regulation

Retrieved on: 
Monday, September 4, 2023
Technology, Medical Devices, Other Technology, Software, Biotechnology, Other Health, Health, General Health, Artificial Intelligence, CE, CT, Radiation, BSI, CPT, Osteoporosis, TBS, Multimedia, BMD, Medical Device Regulation Act, Medical device regulation, Certification, DXA, MDR, X-ray, Patient, Calvarial doughnut lesions-bone fragility syndrome, AI, EEC, FDA, Safety, Medical device, Medical imaging

Medimaps Group, a Swiss/Global med-tech company specializing in image-processing software with AI capabilities for assessing bone health, announced today it received the Medical Device Regulation (MDR) certification from its notified body BSI (CE 2797) for its management system and product portfolio.

Key Points: 
  • Medimaps Group, a Swiss/Global med-tech company specializing in image-processing software with AI capabilities for assessing bone health, announced today it received the Medical Device Regulation (MDR) certification from its notified body BSI (CE 2797) for its management system and product portfolio.
  • By successfully navigating the rigorous requirements of the Medical Device Regulation, our company proves its ability to deliver innovative and reliable solutions that meet the highest regulatory standards for the benefit of patients, healthcare professionals and medical business partners”, said Prof. Didier Hans, co-founder and CEO of Medimaps Group.
  • The certification ensures that medical device manufacturers meet the most stringent quality requirements in order to commercialize their products in Europe.
  • With this certification, Medimaps Group can continue to supply without interruptions certified TBS iNsight™ software at the highest standard to its valued customers in Europe and subsequent markets.

The Hypoparathyroidism Association Announces Patient Enrollment in First Prospective Hypoparathyroidism Natural History Study

Retrieved on: 
Tuesday, May 23, 2023
Columbia University, Seizure, Columbia University Irving Medical Center, Endocrine disease, Chronic kidney disease, Calcification, Urine, EKG, Hypoparathyroidism, Wheeze, DSM-IV codes, Cohort (statistics), Quality of life, Hope, Brain, Arrhythmia, Associate, Marketing, Common, Calcium, PTH, Columbia University Vagelos College of Physicians and Surgeons, Primary familial brain calcification, Gland, Patient, Physician, Cramp, Risk, Blood, Kidney, Vascular disease, Retrospective cohort study, Calvarial doughnut lesions-bone fragility syndrome, Vitamin D, Ageing, Health, Parathyroid hormone, Disease, Dietary supplement, Medical imaging, Nursing, Surgeon, Medicine

NEW YORK, May 23, 2023 (GLOBE NEWSWIRE) -- The Hypoparathyroidism Association today announced patient enrollment in a first-of-its-kind natural history study of hypoparathyroidism, designed to gain insight into the long-term complications of a rare disorder that affects an estimated 77,000 – 115,000 patients in the U.S. The study will be led by Mishaela Rubin, M.D., Associate Professor of Medicine at Columbia University Vagelos College of Physician and Surgeons, and is sponsored by the U.S. Food & Drug Administration’s Office of Orphan Products Development (OOPD).

Key Points: 
  • NEW YORK, May 23, 2023 (GLOBE NEWSWIRE) -- The Hypoparathyroidism Association today announced patient enrollment in a first-of-its-kind natural history study of hypoparathyroidism, designed to gain insight into the long-term complications of a rare disorder that affects an estimated 77,000 – 115,000 patients in the U.S.
  • Common short-term complications of hypoparathyroidism include muscle cramps and cognitive dysfunction, with less common complications including seizures, arrhythmias, and wheezing.
  • As a result of these symptoms, patients living with hypoparathyroidism often have a decreased quality of life.
  • The natural history study will inform understanding of the longer-term effects of hypoparathyroidism and provide insight into the impacts of current disease management practices.

2022 Postmenopausal Osteoporosis Pipeline Insight Report: Featuring PhytoHealth, Luye Pharma, Sandoz International and Addpharma Among Others - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 7, 2023
Health, Pharmaceutical, Route, III, Porosity, Therapy, Incidence, Acquisition, Calvarial doughnut lesions-bone fragility syndrome, Fracture, Teva Pharmaceuticals, Bone, Woman, Administration, NDA, Quality of life, Osteoporosis, Risk, Route of administration, Dietary supplement, Menopause

The "Postmenopausal Osteoporosis - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Postmenopausal Osteoporosis - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • This report provides comprehensive insights about 18+ companies and 18+ pipeline drugs in Postmenopausal osteoporosis pipeline landscape.
  • A detailed picture of the Postmenopausal osteoporosis pipeline landscape is provided which includes the disease overview and Postmenopausal osteoporosis treatment guidelines.
  • Postmenopausal osteoporosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.