Inborn errors of metabolism

AstraZeneca demonstrates commitment to patients living with amyloidosis at the 2024 International Symposium on Amyloidosis (ISA)

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목요일, 5월 23, 2024
Biotechnology, FDA, Pharmaceutical, Health, Patient, RWE, Bortezomib, Science, Heart failure, Prevalence, Diagnosis, ISA, Disease, Epidemiology, EMR, Senior, Death, Alexion, Ionis, Macrophage, Incidence, Polyneuropathy, Cardiomyopathy, Phagocytosis, ATTR, Poster, Inborn errors of metabolism, Outline, AstraZeneca, Daratumumab, Amyloid, Pharmaceutical industry

AstraZeneca and Alexion, AstraZeneca Rare Disease, will showcase 14 studies, including real-world evidence (RWE), from their portfolio and pipeline of investigational amyloidosis therapies at the International Symposium on Amyloidosis (ISA), in Rochester, MN from May 26–30, 2024.

Key Points: 
  • AstraZeneca and Alexion, AstraZeneca Rare Disease, will showcase 14 studies, including real-world evidence (RWE), from their portfolio and pipeline of investigational amyloidosis therapies at the International Symposium on Amyloidosis (ISA), in Rochester, MN from May 26–30, 2024.
  • Sarah Walters, Vice President, US Cardiovascular, Renal & Metabolic Diseases, AstraZeneca, said: “AstraZeneca is dedicated to advancing the science and improving the lives of patients that are impacted by this devastating disease.
  • A retrospective chart analysis from the US EMR TriNetX database will report outcomes in AL amyloidosis patients with renal involvement receiving bortezomib or daratumumab.
  • A comprehensive list of AstraZeneca key abstracts to be presented at the 2024 International Symposium on Amyloidosis includes7:

Arcturus Therapeutics Announces First Quarter 2024 Financial Update and Pipeline Progress

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수요일, 5월 8, 2024
Biotechnology, Infectious Diseases, COVID-19, Health, Pharmaceutical, Society, COVID, Investment, Meiji, ARCT, Ornithine transcarbamylase deficiency, Safety, SARS-CoV-2, Travel, European Commission, Research, OTC, Cystic fibrosis, Lyme disease, RNA, MAA, Inborn errors of metabolism, Trial of the century, Vaccine, CDMO, Omicron, SRR, GMT, SAD, Liver, CSL, EMA, JPMorgan Chase, SIMD, Civil service commission, Infection, Messenger, Arcturus Therapeutics, Therapy, European Medicines Agency, Pharmaceutical industry

“Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.

Key Points: 
  • “Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.
  • To support this effort, Arcturus along with CDMO partners are on track to deliver the initial 4 million commercial doses of Kostaive in Q3.
  • The Company will share a progress update on the Phase 2 study on July 1, 2024.
  • The expected cash runway extends at least three years based on the current pipeline and programs through the first quarter of fiscal year 2027.

Ascendis Pharma Reports First Quarter 2024 Financial Results

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목요일, 5월 2, 2024
FDA, Endocrine gland, Hypoparathyroidism, Element, GHD, Achondroplasia, Commercial, New Drug Application, Biologics license application, Development, Prescription, ASCO, Turner syndrome, PDUFA, Patient, Society, Melanoma, Vision, Inborn errors of metabolism, Pharmaceutical industry

If approved, U.S. launch planned in Q3

Key Points: 
  • If approved, U.S. launch planned in Q3
    — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million
    COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.

JDRF Celebrates Research Award Winners and Recognizes Type 1 Diabetes Research Leaders

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목요일, 4월 18, 2024
Consensus, University, Type 1 diabetes, Diabetes, Person, Risk, Food, Research, JDRF, Immunotherapy, Endocrinology, Retinopathy, Disease, Indiana University, Medicine, Metabolism, Clinical trial, Petah Tikva, Pediatrics, AID, Medical Center, Pediatric endocrinology, Inborn errors of metabolism, Quality of life, Division, Growth, Pharmaceutical industry

NEW YORK, April 18, 2024 /PRNewswire/ -- JDRF, the leading global type 1 diabetes (T1D) research and advocacy organization, proudly presented awards to five outstanding leaders in T1D research whose impact has pushed JDRF's mission forward.

Key Points: 
  • NEW YORK, April 18, 2024 /PRNewswire/ -- JDRF, the leading global type 1 diabetes (T1D) research and advocacy organization, proudly presented awards to five outstanding leaders in T1D research whose impact has pushed JDRF's mission forward.
  • She began her career with a JDRF career development award to support one of her first research projects on insulin pump therapy in very young children with diabetes.
  • The award recognizes leaders and innovators of outstanding clinical and translational T1D research.
  • JDRF Research award recipients were recognized at a ceremony in New York City earlier in April 2024.

embecta-sponsored Educational Symposium and Abstracts at ATTD Highlight Role of Insulin Pumps in the Management of Type 2 Diabetes

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수요일, 3월 6, 2024
Knowledge, Division, Endocrinology, T2D, Inborn errors of metabolism, Metabolism, Education, Treatment, Insulin, Physician, Quality of life, ATTD, Faculty, DNP, University, Type 2 diabetes, EMBC, MDI, Fortezza, Case Western Reserve University, Patient, D.O, Poster, Needlestick injury, Risk, Lipohypertrophy, Obesity, Research, Fortezza da Basso, Time in Europe

Additionally, six embecta-sponsored abstracts focusing on injection and insulin pump therapies have been selected for presentation as scientific posters at ATTD 2024.

Key Points: 
  • Additionally, six embecta-sponsored abstracts focusing on injection and insulin pump therapies have been selected for presentation as scientific posters at ATTD 2024.
  • Entitled “Unlocking the potential of insulin pumps for personalized T2D care,” the symposium aims to highlight the significance of insulin pump therapy among the extensive treatment options available for type 2 diabetes (T2D).
  • The six embecta-sponsored abstracts set for presentation as scientific posters, include robust data about insulin therapy via both pumps and multiple daily injections (MDI).
  • Another poster highlights data showing that in individuals with type 2 diabetes, insulin pump therapy eases constraints imposed by diabetes management and improves quality of life.

J.P. Morgan Life Sciences Private Capital Team Adds Healthcare Veteran and Past Seagen CEO David Epstein to Advisory Board

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화요일, 2월 27, 2024
Rubius, Odyssey, Senior, Inborn errors of metabolism, Investment, Drug development, Growth, Sale, IPO, University, Intuitive Surgical, Neurodegenerative disease, Analog Devices, National academy, J. P. Morgan, MIT, Genetic distance, American Philosophical Society, Business development, Journal, Therapy, Imatinib, SRL Diagnostics, MidOcean Partners, Distinction, Laboratory, Cornell University, Drug discovery, Weill Cornell Medicine, Provost, Panasonic Executive Partner, Abbott Laboratories, Flagship, Novartis, Medtronic, MACP, Harvard Medical School, Nilotinib, American Academy, Yale School of Medicine, JPMorgan Chase, AI, MD, Flagship Pioneering, Harvard University, MassMutual, Executive, Cancer, Management

NEW YORK, Feb. 27, 2024 /PRNewswire/ -- J.P. Morgan Life Sciences Private Capital today announced the addition of David Epstein to its Healthcare Advisory Board.

Key Points: 
  • NEW YORK, Feb. 27, 2024 /PRNewswire/ -- J.P. Morgan Life Sciences Private Capital today announced the addition of David Epstein to its Healthcare Advisory Board.
  • Mr. Epstein will now advise the J.P. Morgan Life Sciences Private Capital team.
  • The J.P. Morgan Life Sciences Private Capital team leverages JPMorgan Chase's scale, resources, data assets and healthcare expertise and sits within J.P. Morgan Private Capital, a venture and growth equity investment platform that is part of J.P. Morgan Asset Management.
  • The platform finances the continued growth of private companies and taps into significant pre-IPO value creation opportunities in the consumer and technology, and life sciences sectors.

National Academy of Medicine Member New President of The Obesity Society

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목요일, 2월 1, 2024
Bias, The Obesity Society, Medical school, Growth, University, Metabolism, Science, Obesity, Duke University Hospital, Division, Society, National academy, Patient, Inborn errors of metabolism, Columbia University, Wellness, Executive Committee, ARD, Obesity medicine, Harvard Medical School, Education, Health equity, Doctor of Philosophy, Health, Cornier, Cedars-Sinai Medical Center, Clinical research, Internal medicine

ROCKVILLE, Md. , Feb. 1, 2024 /PRNewswire/ -- A member of the National Academy of Medicine is the new president of The Obesity Society (TOS), the organization announced today.

Key Points: 
  • , Feb. 1, 2024 /PRNewswire/ -- A member of the National Academy of Medicine is the new president of The Obesity Society (TOS), the organization announced today.
  • He also co-directs the Atrium Health Wake Forest Baptist Weight Management Center, where he oversees medical weight management programs.
  • "I am honored to serve as president of TOS during such a transformative time in the field of obesity.
  • The Obesity Society (TOS) is the leading organization of scientists and health professionals devoted to understanding and reversing the epidemic of obesity and its adverse health, economic and societal effects.

MGI Empowers the Completion of Nearly 60,000 Samples for The Million Microbiome of Humans Project

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월요일, 5월 15, 2023
University College London, Karolinska Institute, DNA, Disease, University, Partnership, Institute of technology, Food, COVID-19, INRAE, Research, Biomedical Research, DNB, USD, De novo, Organization, Inborn errors of metabolism, United Nations Research Institute for Social Development, Technical University of Denmark, Cancer, Medicine, Copenhagen, University of Copenhagen Faculty of Health and Medical Sciences, Ecology, Health, Metagenomics, Microbiology, MGI, Virulence, Fine chemical, Pharmaceutical industry, Antibiotics, Environment

Despite a brief interruption by the COVID-19 pandemic, we are delighted to see such a monumental milestone merely four years into the project."

Key Points: 
  • Despite a brief interruption by the COVID-19 pandemic, we are delighted to see such a monumental milestone merely four years into the project."
  • Advances in genome sequencing has enabled researchers to better characterize the composition of the microbiome through identification of unculturable microbes.
  • Besides human microbiome, it is highly applicable in agricultural microbiome studies, environmental microbiome studies, pathogen surveillance and identification, and monitoring of antimicrobial resistance genes.
  • MGISP-960's fully automatic operation design allows DNA extraction of 50,000 samples per year and library preparation of 25,000 samples per year.

Dalriada further strengthens small molecule discovery expertise with appointment of veteran Dr. Frosty Loechel as SVP Biology, Discovery Strategy group

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수요일, 4월 19, 2023
Lundbeck, Dál Riata, CNS, Inborn errors of metabolism, Thermo Fisher Scientific, BioImage, Drug discovery, Biology, SVP, Pharmacology, Pharmaceutical industry

TORONTO, April 19, 2023 (GLOBE NEWSWIRE) -- Dalriada, a leading Canadian contract research organization specializing in small molecule therapies, announces today the appointment of Frosty Loechel, Ph.D. as SVP Biology, Discovery Strategy group.

Key Points: 
  • TORONTO, April 19, 2023 (GLOBE NEWSWIRE) -- Dalriada, a leading Canadian contract research organization specializing in small molecule therapies, announces today the appointment of Frosty Loechel, Ph.D. as SVP Biology, Discovery Strategy group.
  • The appointment confirms Dalriada’s ambition to remain at the forefront of expertise and capabilities in small molecule drug discovery with its pioneering and agile TURN-KEY™ model.
  • At Dalriada, Dr. Loechel will be responsible for scientific strategy in biology for drug discovery programs.
  • Welcoming Dr. Loechel to the Company, Dalriada’s Chief Executive and co-founder, Dr. Diana Kraskouskaya commented: “I am delighted to have Dr. Loechel join Dalriada.

Premier Research Fortifies Cell and Gene Therapy Leadership Team, Strengthening End-to-End Capabilities in Complex Asset Development

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월요일, 4월 17, 2023
Pharmacy, Central nervous system, CGT, Gene, Quality by design, Pediatrics, CMC, NMES, Strategic planning, RNA, CNS, DSM-IV codes, Metabolism, Inborn errors of metabolism, Dermatology, Documentation, FACMG, Aloba, Hematology, Regulatory affairs, Cardiology, Research, TGA, FDA, Neurology, Meyer, Safety, Medical device, Pharmaceutical industry, EU, Cell

MORRISVILLE, N.C., April 17, 2023 /PRNewswire/ -- Premier Research announced today the addition of Kenneth Ndugga-Kabuye, M.D., FACMG, as VP, Cell & Gene Therapy to the strategic Cell & Gene Therapy (CGT) Leadership Team. Ndugga-Kabuye joins experts Jessica Merryfield, Executive Director, Cell & Gene Therapy, Olu Aloba, Ph.D., VP, CMC Services and Greg Meyer, VP, Regulatory Affairs in addressing the complexities of CGT development, as evolving regulatory receptivity creates a supportive environment for innovation.

Key Points: 
  • Cell and Gene Therapy Expert Dr. Kenneth Ndugga-Kabuye to Lead
    Clinical Strategy With Collaboration From Operations, CMC and Regulatory Experts
    MORRISVILLE, N.C., April 17, 2023 /PRNewswire/ -- Premier Research announced today the addition of Kenneth Ndugga-Kabuye, M.D., FACMG, as VP, Cell & Gene Therapy to the strategic Cell & Gene Therapy (CGT) Leadership Team.
  • "The speed at which agency guidance is changing, coupled with complicated cell and gene therapy study logistics, is creating demand for early, in-depth strategic planning," Premier Research Chief Operating Officer Michael Arlotto, Ph.D., said.
  • "Cell and gene therapy approval pathways are highly variable among regulatory agencies globally," Ndugga-Kabuye said.
  • Jessica Merryfield, Executive Director, Cell & Gene Therapy, who has more than 20 years of clinical research experience, including more than 10 years specializing in CGT.