Congenital myasthenic syndrome

argenx Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Thursday, May 9, 2024
MuSK protein, FDA, ARDA, IND, PDUFA, Medicine, Autoimmunity, Efgartigimod alfa, Glomerulonephritis, CMS, Inflammation, ITP, IIP, ALS, Alfa Romeo, C2, Therapy, Systemic scleroderma, Safety, Euronext, Nektar, DGF, Sjögren syndrome, CIDP, Biology, IL-6, Nektar Therapeutics, GMG, TED, File, MMN, Lupus nephritis, Kidney, AMR, Multifocal motor neuropathy, Patient, Congenital myasthenic syndrome, Immune system, Dermatomyositis, Growth, Amgen, Pharmaceutical industry

“The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • “We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy.
  • VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

argenx Reports Full Year 2023 Financial Results and Provides Fourth Quarter Business Update

Retrieved on: 
Thursday, February 29, 2024
DGF, Neonatal Fc receptor, ALS, Congenital myasthenic syndrome, CMS, CIDP, MMN, Alfa Romeo, IIP, IL-6, FDA, Inflammation, MHLW, PDUFA, Euronext, Neurology, MuSK protein, Kidney, ARDA, Health care, C2, GMG, Ministry of Health, Dermatomyositis, Patient, Efgartigimod alfa, MHRA, Medicine, Multifocal motor neuropathy, Immune system, Pharmaceutical industry

“argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart.
  • Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Monday, January 8, 2024
Immunoglobulin therapy, GMG, FDA, PRV, Congenital myasthenic syndrome, ARDA, Research Councils UK, Euronext, Inflammation, Cohort, PGIC, NRDL, Autoimmunity, Efgartigimod alfa, Dermatomyositis, Conference, MuSK protein, IND, CIDP, Pharmacokinetics, MMN, Safety, Therapy, Patient, ALS, Immune system, IL-6, Medicine, International Myeloma Foundation, IIP, FSS, Neonatal Fc receptor, Multifocal motor neuropathy, Growth, Pharmaceutical industry

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

Neogen® Unveils Over 100 New Genetic Tests

Retrieved on: 
Tuesday, August 8, 2023
Genetics, Alaskan Malamute, Veterinarian, Deafness, King Hamad University of Nursing and Associated Medical Sciences, Animal, Congenital myasthenic syndrome, Scent hound, Health, Neogen, Disease, Vaccine, Pharmaceutical industry

"These new tests can identify genetic sequences associated with potential diseases and other traits and can provide invaluable insights into a canine's genetic composition," said Dr. Robert Westra, Associate Medical Director at Neogen.

Key Points: 
  • "These new tests can identify genetic sequences associated with potential diseases and other traits and can provide invaluable insights into a canine's genetic composition," said Dr. Robert Westra, Associate Medical Director at Neogen.
  • Dedicated to the continuous advancement of canine health and wellbeing, Paw Print Genetics has introduced 49 new tests, increasing its full testing portfolio to nearly 350 breed-specific tests.
  • Results are reported as normal, carrier, or at-risk/affected, which can provide extensive information about a dog's genetic makeup.
  • Paw Print Genetics and Canine HealthCheck tests are available for purchase starting today on the Paw Print Genetics and Canine Health Check websites.

argenx Reports Half Year 2023 Financial Results and Provides Second Quarter Business Update

Retrieved on: 
Thursday, July 27, 2023
Congenital myasthenic syndrome, CIDP, Efgartigimod alfa, MuSK protein, ALS, SC, GMG, C2, Euronext, Patient, Risk, Immunoglobulin G, Growth, Ecosystem, Neonatal Fc receptor, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Pharmaceutical industry, EU

VYVGART® Hytrulo now available in the U.S. with first vials shipped in July

Key Points: 
  • VYVGART® Hytrulo now available in the U.S. with first vials shipped in July
    Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its half year 2023 financial results and provided a second quarter business update.
  • “We are thrilled to see the momentum continue across all aspects of our business, with a catalyst-rich first half of the year.
  • Our ambition level is high and we are positioning argenx for long-term growth with VYVGART, empasiprubart, and our pipeline of immunology solutions.
  • argenx expects to nominate one new pipeline candidate in 2023.

Amplo Biotechnology Secures Fast Track STTR Funding for AMP-201, a Promising Gene Therapy Targeting Collagen Q Deficiency

Retrieved on: 
Wednesday, May 31, 2023
University of California, Davis, Congenital myasthenic syndrome, Collagen, Biotechnology, CMS, Doctor of Philosophy, Clinical trial, Quality of life, Fast Track, MBA, Nagoya University, AAV, Patient, COLQ, SAN, NIH, Safety, Pharmaceutical industry

This highly competitive grant will provide substantial funding for the advancement of AMP-201, an AAV-ColQ gene therapy designed to address the severe congenital myasthenic syndrome (CMS) caused by Collagen Q (ColQ) Deficiency.

Key Points: 
  • This highly competitive grant will provide substantial funding for the advancement of AMP-201, an AAV-ColQ gene therapy designed to address the severe congenital myasthenic syndrome (CMS) caused by Collagen Q (ColQ) Deficiency.
  • Under an exclusive licensing agreement, Amplo Biotechnology has gained access to AAV-ColQ data developed by Professor Kinji Ohno's laboratory at Nagoya University, which forms the basis of AMP-201.
  • In the Fast Track STTR project, Amplo will collaborate with Professor Ricardo Maselli, a CMS expert neurologist and researcher at UC-Davis.
  • Amplo Biotechnology's development of AMP-201 builds upon it's progression of AMP-101, an AAV-DOK7 gene therapy targeting Dok-7 congenital myasthenic syndrome, which Amplo expects to bring to clinical trial in 2024.

argenx Reports First Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, May 4, 2023
Congenital myasthenic syndrome, CIDP, Efgartigimod alfa, University, MuSK protein, IQVIA, GMG, SC, Health care, COVID-19, ITP, Cancer, Postural orthostatic tachycardia syndrome, MMN, Ministry, Euronext, ALPHA, Patient, UCHealth, PDUFA, RHO, Ecosystem, POC, Neonatal Fc receptor, FC, MHRA, Pharmaceutical industry, Vaccine, EU, Health

88 events achieved in ADHERE trial; topline data now expected in July 2023

Key Points: 
  • 88 events achieved in ADHERE trial; topline data now expected in July 2023
    Enrollment completed in ADVANCE-SC and ADDRESS trials; topline data from both expected in fourth quarter of 2023
    Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its first quarter 2023 financial results and provided a business update.
  • “Throughout the first quarter, we made significant progress advancing our mission to redefine what well-controlled means in the treatment of autoimmune diseases.
  • By the end of 2023, efgartigimod is expected to be approved, in regulatory review or in development in 13 severe autoimmune diseases.
  • argenx expects to nominate one new development candidate in 2023.

Antibody Therapy Rescues Mice from Lethal Nerve-Muscle Disease

Retrieved on: 
Wednesday, June 23, 2021
Medical specialties, Immunology, Branches of biology, Antibody, Glycoproteins, Congenital myasthenic syndrome, Clinical medicine

Led by NYU Grossman School of Medicine researchers, a new study found that an antibody treatment not only rescued young mice from a form of congenital myasthenia (CM) but also reversed disease relapse in adult mice.

Key Points: 
  • Led by NYU Grossman School of Medicine researchers, a new study found that an antibody treatment not only rescued young mice from a form of congenital myasthenia (CM) but also reversed disease relapse in adult mice.
  • "To our knowledge, our study is the first to fully counter a lethal congenital flaw by targeted therapy, restoring synapse formation with an antibody that encourages the action of a protein downstream of the mutant gene," says lead author Steven J.
  • Further, when the team withdrew the antibody treatment, adult mice relapsed and displayed movement problems, which were reversed by re-starting antibody treatment.
  • 9,329,182, titled "Method of treating motor neuron disease with an antibody that agonizes MuSK", while Burden, Shohei Koide, Akiko Koide, Oury, and Leloup are inventors on a patent application no.