X-linked hypophosphatemia

Kyowa Kirin Presents Real-World Findings at ASBMR Annual Meeting Highlighting Progressive Disease Burden of X-linked Hypophosphatemia (XLH)

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Monday, October 16, 2023
Komodo, Diagnosis, Enthesopathy, Osteoarthritis, MBA, Health care, Physician, Hypertension, Obesity, Research, Calcitriol, Myalgia, Burosumab, Prevalence, Tendon, X-linked hypophosphatemia, XLH, Society, Kyowa Kirin, TSE, Spinal stenosis, Health, American Society for Bone and Mineral Research, Arthralgia, Fracture, Disease, Medicare, Kidney, Depression, Patient, DSM-IV codes, Kidney disease, Commercial, FH, Muscle weakness, Pharmaceutical industry

Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.

Key Points: 
  • Most patients had received some form of conventional therapy for XLH prior to burosumab treatment, with 61% receiving calcitriol and 43% receiving phosphate supplements.
  • Older patients were under-represented in the study population, limiting the generalizability of the findings to this group.
  • In this retrospective observational cohort study, researchers examined healthcare claims data from the Merative™ MarketScan® Commercial and Medicare Databases.
  • The differences between the study groups were apparent even among young children and persisted throughout a person's life.

Kyowa Kirin to Present New Research on X-linked Hypophosphatemia (XLH) at ASBMR Annual Meeting

Retrieved on: 
Thursday, October 12, 2023
Diagnosis, MBA, Research, X-linked hypophosphatemia, XLH, Society, Kyowa Kirin, TSE, American Society for Bone and Mineral Research, Disease, Adolescence, Poster, Life, Patient, International, Pharmaceutical industry

PRINCETON, N.J., Oct. 12, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company based in Japan, will share new research that advances understanding about the real-world experiences and clinical characteristics of children, adolescents, and adults living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease.

Key Points: 
  • PRINCETON, N.J., Oct. 12, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company based in Japan, will share new research that advances understanding about the real-world experiences and clinical characteristics of children, adolescents, and adults living with X-linked hypophosphatemia (XLH), a rare genetic metabolic bone disease.
  • The findings will be presented in a series of poster presentations at the American Society for Bone and Mineral Research (ASBMR) 2023 annual meeting, which takes place October 13-16 in Vancouver, BC, Canada.
  • "Individuals living with XLH face a lifelong struggle of managing their disease and progressively worsening morbidities, which negatively impact their life," said Zhiyi Li, MA, MBA, Director of Health Economics and Outcomes Research at Kyowa Kirin North America.
  • "The breadth of the research we are presenting at ASBMR reflects our commitment to helping inform a more complete understanding of XLH and its' management."

The Shriners Hospitals for Children - Canada Research Centre celebrates half a century of existence

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Thursday, August 17, 2023
Dentistry, Parent, Teaching, Anxiety, Research institute, Quality of life, Bone, Collagen, type I, alpha 1, OC, Research, Medicine, Bone disease, Health, Doctor of Philosophy, Government, Pain, DSM-IV codes, International Clinical Research Center, Metabolic bone disease, Student, MD, Physician, Shriners Hospitals for Children, McGill University, Virtual reality, Knowledge, Diagnosis, McGill University Health Centre, X-linked hypophosphatemia, Nursing research, MRC Human Genetics Unit, Shriners, Hormone replacement therapy, Shaukat Khanum Memorial Cancer Hospital and Research Centre, Nursing, Child

So research into mineralized tissue has always been a strength in Quebec," explains Professor René St-Arnaud, PhD, Director of the Research Center.

Key Points: 
  • So research into mineralized tissue has always been a strength in Quebec," explains Professor René St-Arnaud, PhD, Director of the Research Center.
  • In 1973, following the integration of Shriners Hospitals for Children – Canada into the Quebec health care network, Shriners Children’s management, with the approval of the provincial government, created the first Research Centre within its hospital network.
  • The Shriners Hospital for Children - Canada Research Centre is constantly developing new ways to care for children.
  • To mark this 50th anniversary at Shriners Hospital for Children – Canada, Dr. St-Arnaud hosted a scientific seminar yesterday featuring a number of prestigious speakers.

Kyowa Kirin North America Assumes Commercial Leadership Role for CRYSVITA® (burosumab-twza) Injection in North America

Retrieved on: 
Thursday, April 27, 2023
Burosumab, Hypophosphatemia, Education, Pledge, Ultragenyx, Research, Kyowa Kirin, Patient, TIO, FGF23, Neoplasm, Rare, X-linked hypophosphatemia, XLH, TSE, Pharmaceutical industry

BEDMINSTER, N.J., April 27, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a top 50 global specialty pharmaceutical company, today assumes primary commercial leadership for CRYSVITA® (burosumab-twza) injection in the United States and Canada from Ultragenyx Pharmaceutical, Inc., a transition planned for from the start of their decade-long collaboration.

Key Points: 
  • BEDMINSTER, N.J., April 27, 2023 /PRNewswire/ -- Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a top 50 global specialty pharmaceutical company, today assumes primary commercial leadership for CRYSVITA® (burosumab-twza) injection in the United States and Canada from Ultragenyx Pharmaceutical, Inc., a transition planned for from the start of their decade-long collaboration.
  • The transition builds on the company's existing commercial portfolio and commitment to address the needs of people living with rare diseases.
  • Their contributions were instrumental in expediting late-stage trials, securing regulatory approval, and delivering treatment to thousands of patients," said Steve Schaefer, President, Kyowa Kirin North America.
  • To prepare for its new commercial leadership role in North America, Kyowa Kirin has engaged with community leaders and people living with XLH and TIO to gain perspective on their experiences and how best to support them.

Mereo BioPharma Announces Appointment of Dr. Abdul Mullick to Board of Directors

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Tuesday, May 17, 2022
BOS, GCC1, Osteogenesis imperfecta, Company, Disease, Blood, OI, Neoplasm, X-linked hypophosphatemia, U.S. Securities and Exchange Commission, Genzyme, Sanofi, Doctor of Philosophy, Molecular biology, Leadership, Monaco Age Oncologie, Patient, NASDAQ, Vifor Pharma, KKI, Degenerative disease, Marketing, Respiratory tract, Novartis, Form 6-K, Forward-looking statement, Board, Ovarian cancer, Head, Therapy, Security (finance), University of Bristol, Annual report, AATD, GLOBE, Pharmaceutical industry

LONDON and REDWOOD CITY, Calif., May 17, 2022 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (Mereo or the Company), a clinical-stage biopharmaceutical company focused on oncology and rare diseases, today announced the appointment of Dr. Abdul Mullick to the Companys Board of Directors, effective immediately.

Key Points: 
  • LONDON and REDWOOD CITY, Calif., May 17, 2022 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (Mereo or the Company), a clinical-stage biopharmaceutical company focused on oncology and rare diseases, today announced the appointment of Dr. Abdul Mullick to the Companys Board of Directors, effective immediately.
  • We are very excited to welcome Abdul to the Board at such a pivotal time for the Company, said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo.
  • I believe Abdul will be an asset to the Board and very much look forward to working with him.
  • Mereo has shown outstanding commitment to helping underserved patient populations and I am honored to be joining the Board, added Dr. Mullick.

SiSaf Announces Michael J. Econs MD, World-leading Expert in Metabolic Bone Disease, as Clinical Advisor

Retrieved on: 
Thursday, January 27, 2022
Osteoclast, Therapy, Trial of the century, Patient, Knowledge, RNA, Bone, Neoplasm, XLH, Lists of diseases, Metabolism, Lipid, Research, Silicon, Gamma ray, X-linked hypophosphatemia, Indiana University School of Medicine, Economics, CEO, Osteomalacia, Vickers Venture Partners, Company, Genetic distance, Interferon, IND, Autosome, MD, Disease, CLCN7, Metabolic bone disease, Pharmaceutical industry, Vaccine, Medical imaging, Medicine, Endocrinology, FGF23

GUILDFORD, England, Jan. 27, 2022 /PRNewswire/ -- SiSaf Ltd, a company developing RNA therapeutics for rare genetic skeletal disorders, today announced that it has appointed Professor Michael J. Econs, MD as Clinical Advisor.

Key Points: 
  • GUILDFORD, England, Jan. 27, 2022 /PRNewswire/ -- SiSaf Ltd, a company developing RNA therapeutics for rare genetic skeletal disorders, today announced that it has appointed Professor Michael J. Econs, MD as Clinical Advisor.
  • Although this did not show clinical benefit , the experience gained will be invaluable in guiding SiSaf in its clinical studies."
  • SiSaf is using these next-generation lipid nanoparticles to advance an in-house pipeline of RNA therapeutics for rare genetic skeletal disorders.
  • SiSaf is a private company supported by venture capital investors including Vickers Venture Partners & UK Future Fund.

SiSaf Announces Michael J. Econs MD, World-leading Expert in Metabolic Bone Disease, as Clinical Advisor

Retrieved on: 
Thursday, January 27, 2022
Osteoclast, Therapy, Trial of the century, Patient, Knowledge, RNA, Bone, Neoplasm, XLH, Lists of diseases, Metabolism, Lipid, Research, Silicon, Gamma ray, X-linked hypophosphatemia, Indiana University School of Medicine, Economics, CEO, Osteomalacia, Vickers Venture Partners, Company, Genetic distance, Interferon, IND, Autosome, MD, Disease, CLCN7, Metabolic bone disease, Pharmaceutical industry, Vaccine, Medical imaging, Medicine, Endocrinology, FGF23

GUILDFORD, England, Jan. 27, 2022 /PRNewswire/ -- SiSaf Ltd, a company developing RNA therapeutics for rare genetic skeletal disorders, today announced that it has appointed Professor Michael J. Econs, MD as Clinical Advisor.

Key Points: 
  • GUILDFORD, England, Jan. 27, 2022 /PRNewswire/ -- SiSaf Ltd, a company developing RNA therapeutics for rare genetic skeletal disorders, today announced that it has appointed Professor Michael J. Econs, MD as Clinical Advisor.
  • Although this did not show clinical benefit , the experience gained will be invaluable in guiding SiSaf in its clinical studies."
  • SiSaf is using these next-generation lipid nanoparticles to advance an in-house pipeline of RNA therapeutics for rare genetic skeletal disorders.
  • SiSaf is a private company supported by venture capital investors including Vickers Venture Partners & UK Future Fund.

Long-Term CRYSVITA® ▼ (burosumab) Treatment Reduces the Burden of Disease in Adults With X-Linked Hypophosphataemia (XLH), a Rare Genetic Metabolic Bone Disease

Retrieved on: 
Friday, September 24, 2021
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, People, Immunoglobulin G, Burosumab, Swissmedic, RMD, PRO, Human musculoskeletal system, Ministry, Kirin, Kyowa Hakko Kirin, Serum, Fatigue, Calcium, Ministry of Health, Labour and Welfare, Fracture, NASDAQ, List of health departments and ministries, Patient, Engineering, BMJ, Parallel Walk Test, Adult, Vitamin D, Urine, Kidney, Phosphate, Royal commission, XLH, Lists of diseases, Osteomalacia, Life, Pain, Physical, X-linked hypophosphatemia, Nephrology, BFI, Neurology, Hypophosphatemia, Research, Asia, Data, FGF23, European Commission, NHI, Safety, National health insurance, Quality of life, Bone disease, WOMAC, Muscle, Rheumatology, Pharmaceutical industry, Dietary supplement, Phosphorus, X-linked hypophosphataemia, CRYSVITA® (burosumab), Kyowa Kirin, X-LINKED HYPOPHOSPHATAEMIA, CRYSVITA® (BUROSUMAB), KYOWA KIRIN

Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) today announced the publication of new data highlighting the sustained benefits of treatment with CRYSVITA (burosumab) in adults with X-linked hypophosphataemia (XLH), a rare genetic metabolic bone disease.

Key Points: 
  • Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin) today announced the publication of new data highlighting the sustained benefits of treatment with CRYSVITA (burosumab) in adults with X-linked hypophosphataemia (XLH), a rare genetic metabolic bone disease.
  • The data show that adults with XLH experience substantial pain, stiffness, fatigue and impairment in physical and ambulatory function.
  • Burosumab treatment has previously been shown to improve phosphate homeostasis in adult XLH patients, compared to placebo.
  • One of our areas of focus is to generate new data that improve our understanding of how best to manage and treat XLH.

Kyowa Kirin Announces EU Approval for the Self-administration of CRYSVITA® ▼ (burosumab) to Treat X-Linked Hypophosphataemia (XLH)

Retrieved on: 
Monday, July 19, 2021
Radiology, Pharmaceutical, Genetics, Health, Health, Monoclonal antibodies, Orphan drugs, Breakthrough therapy, Burosumab, X-linked hypophosphatemia, Kyowa Hakko Kirin, Kirin, Health care

The self-administration of CRYSVITA is at the discretion of a treating physician, providing those self-administering are competent and comfortable doing so.

Key Points: 
  • The self-administration of CRYSVITA is at the discretion of a treating physician, providing those self-administering are competent and comfortable doing so.
  • Abdul Mullick, President of Kyowa Kirin International, said: This approval is another significant milestone for the treatment of people living with XLH.
  • Kyowa Kirin is committed to improving the lives of patients and their families by giving healthcare professionals better ways to care for them.
  • By gaining EU approval for the self-administration of CRYSVITA, we have created another valuable option in the care of children, adolescents and adults with XLH.

Inozyme Pharma Publishes Data Supporting INZ-701 as a Potential Treatment for ENPP1 Deficiency

Retrieved on: 
Thursday, April 29, 2021
Generalized arterial calcification of infancy, Ectonucleotide pyrophosphatase/phosphodiesterase 1, Rare diseases, Health, Clinical medicine, Metabolic disorders, Medicine, X-linked hypophosphatemia

These results strengthen the rationale for INZ-701 as a potential treatment for ENPP1 Deficiency and support the endpoints for our planned clinical trials,\xe2\x80\x9d said Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma.

Key Points: 
  • These results strengthen the rationale for INZ-701 as a potential treatment for ENPP1 Deficiency and support the endpoints for our planned clinical trials,\xe2\x80\x9d said Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma.
  • \xe2\x80\x9cWe are pleased that the editors of the JBMR recognize the importance of these preclinical data and their potential implications for patients and families affected by ENPP1 Deficiency.\xe2\x80\x9d\nENPP1 Deficiency is a progressive condition that manifests as a spectrum of disease.
  • ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2).
  • The Company anticipates that subsequent events and developments will cause the Company\xe2\x80\x99s views to change.