Sex linkage

ADMA Biologics Announces Third Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, November 9, 2022
Wiskott–Aldrich syndrome, Increase, Transplant, Risk, Sale, Infection, Virus, Company, Conference, San Jose BioCenter, 8-K, Review, Immunity, Forward-looking statement, Plasma, Morgan Stanley, Marketing, RSV, Environment, IVIG, SEC, Immunodeficiency, Collection, U.S. Securities and Exchange Commission, Research, Patient, Private Securities Litigation Reform Act, Protein, PIDD, Hepatitis B virus, Nasdaq, Food and Drug Administration, Antibody, European Patent Office, Bacteria, Growth, FDA, Income, GLOBE, Respiratory syncytial virus, Sex linkage, PIN, Investment, Federation of Clinical Immunology Societies, Publication, Disease, General manager, ADMA, Urinary tract infection, Iron ore, Pharmaceutical industry, Video game, Vaccine, Medical device, Reinsurance, Air cargo, PI

ET

Key Points: 
  • ET
    RAMSEY, N.J. and BOCA RATON, Fla., Nov. 09, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing, and developing specialty plasma-derived biologics, today announced its third quarter 2022 financial results and provided a business update.
  • Third Quarter 2022 and Recent Achievements:
    Significant Revenue Growth: Achieved third quarter 2022 total revenues of $41.1 million, as compared to $20.7 million during the third quarter of 2021, an increase of $20.4 million, or approximately 99%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong year-to-date execution, ADMA anticipates total 2022 revenues will reach approximately $145 million.
  • Third Quarter 2022 Financial Results:
    Total revenues for the third quarter ended September 30, 2022 were $41.1 million, as compared to $20.7 million during the third quarter of 2021, an increase of $20.4 million, or approximately 99%.

Fabry Disease Market to Boost at a CAGR of 7.65% by 2032 | DelveInsight

Retrieved on: 
Tuesday, October 4, 2022
ACE, GB3, Amicus, Hearing loss, Glycogen storage disease, Vertigo, Fabry disease, Lysosome, Disease, Prevalence, Man, Analgesic, Pain, Enzyme replacement therapy, ERT, Therapy, ADA, Overalls, Genetically modified bacteria, Headache, Sangamo Therapeutics, Patient, Sex linkage, United Kingdom, Tissue, Risk, Woman, FD, Population, Pharmaceutical industry, Lithium, Vaccine, Protalix BioTherapeutics, Fabry, Takeda, Alpha-galactosidase

DelveInsight's Fabry Disease Market Insights report includes a comprehensive understanding of current treatment practices, Fabry disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].

Key Points: 
  • DelveInsight's Fabry Disease Market Insights report includes a comprehensive understanding of current treatment practices, Fabry disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].
  • The increasing prevalence of the disease, along with the promising emerging pipeline therapies with new mechanisms of action, will fuel the Fabry disease market during the forecast period.
  • In the European and Japanese Fabry disease treatment market, Replagal (Takeda/Shire), Fabrazyme (Sanofi-Genzyme), and Galafold (Amicus Therapeutics) are currently the only approved therapies for Fabry disease patients.
  • To know more about Fabry disease medication, visit @ Fabry Disease Treatment Market

Fabry Disease Market to Boost at a CAGR of 7.65% by 2032 | DelveInsight

Retrieved on: 
Tuesday, October 4, 2022
ACE, GB3, Amicus, Hearing loss, Glycogen storage disease, Vertigo, Fabry disease, Lysosome, Disease, Prevalence, Man, Analgesic, Pain, Enzyme replacement therapy, ERT, Therapy, ADA, Overalls, Genetically modified bacteria, Headache, Sangamo Therapeutics, Patient, Sex linkage, United Kingdom, Tissue, Risk, Woman, FD, Population, Pharmaceutical industry, Lithium, Vaccine, Protalix BioTherapeutics, Fabry, Takeda, Alpha-galactosidase

DelveInsight's Fabry Disease Market Insights report includes a comprehensive understanding of current treatment practices, Fabry disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].

Key Points: 
  • DelveInsight's Fabry Disease Market Insights report includes a comprehensive understanding of current treatment practices, Fabry disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].
  • The increasing prevalence of the disease, along with the promising emerging pipeline therapies with new mechanisms of action, will fuel the Fabry disease market during the forecast period.
  • In the European and Japanese Fabry disease treatment market, Replagal (Takeda/Shire), Fabrazyme (Sanofi-Genzyme), and Galafold (Amicus Therapeutics) are currently the only approved therapies for Fabry disease patients.
  • To know more about Fabry disease medication, visit @ Fabry Disease Treatment Market

Timber Pharmaceuticals Receives Positive Opinion on Orphan Designation from European Medicines Agency for TMB-001 for Treatment of Autosomal Recessive Congenital Ichthyosis

Retrieved on: 
Wednesday, September 14, 2022
Securities Exchange Act of 1934, CMC, Safety, NYSE, FDA, COMP, CI, Food, Drug Quality and Security Act, European Medicines Agency, Lumber, Isotretinoin, Nature, Company, European Commission, Genta (company), Sex linkage, Intellectual property, Degenerative disease, Securities Act of 1933, Industry, U.S. Securities and Exchange Commission, Patient, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ASCEND, X-linked ichthyosis, Annual report, Private Securities Litigation Reform Act, XLRI, Committee, Pharmacokinetics, Ichthyosis, GLOBE, Royal commission, Union, Marketing, Security (finance), EMA, Chemistry, Pharmaceutical industry, EU, ARCI

BASKING RIDGE, NJ, Sept. 14, 2022 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the “Company”) (NYSE American: TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced that the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) issued a positive opinion on the Company’s application for orphan designation for TMB-001 for the treatment of autosomal recessive congenital ichthyosis (ARCI). Based on the COMP’s opinion, the European Commission is expected to issue its decision on the orphan designation in approximately 30 days.

Key Points: 
  • Based on the COMPs opinion, the European Commission is expected to issue its decision on the orphan designation in approximately 30 days.
  • I want to thank our team at Timber and all trial investigators and patients who have helped us reach this stage in development.
  • As we wait for the European Commission to issue its decision to hopefully grant orphan designation for TMB-001 in ARCI, Timber now plans to also submit an application for EMA orphan designation for TMB-001 for the treatment of X-linked ichthyosis.
  • Timber Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases.

ADMA Biologics Announces Second Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, August 10, 2022
Shift, U.S. Securities and Exchange Commission, GLOBE, SEC, Patent, Method, Marketing, Patient, PIN, European Patent Office, Infection, Urinary tract infection, Collection, Disease, Metapneumovirus, Sex linkage, Risk, Private Securities Litigation Reform Act, Environment, Respiratory syncytial virus, Increase, Conference, Immunity, San Jose BioCenter, Forward-looking statement, Growth, Income, General manager, ADMA, 8-K, Lists of diseases, IVIG, Morgan Stanley, Coronavirus, Virus, FDA, Antibody, Immunodeficiency, RSV, PIDD, Immunoglobulin therapy, Plasma, Company, Bacteria, Wiskott–Aldrich syndrome, United States Patent and Trademark Office, Protein, Hepatitis B virus, Food and Drug Administration, Adoption, Video game, Renewable energy, Vaccine, Medical device, Tutoring, PI

ET

Key Points: 
  • ET
    RAMSEY, N.J. and BOCA RATON, Fla., Aug. 10, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing, and developing specialty plasma-derived biologics, today announced its second quarter 2022 financial results and provided a business update.
  • Second Quarter 2022 and Recent Achievements:
    Significant Revenue Growth: Achieved second quarter 2022 total revenues of $33.9 million, as compared to $17.8 million during the second quarter of 2021, an increase of $16.1 million, or approximately 90%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong start to the year, ADMA anticipates total 2022 revenues will exceed $130 million.
  • Second Quarter 2022 Financial Results:
    Total revenues for the second quarter ended June 30, 2022 were $33.9 million, as compared to $17.8 million during the second quarter of 2021, an increase of $16.1 million, or approximately 90%.

GenSight Biologics Announces Publication of a Study of the Impact of LHON Disease on the Lives of Patients and Relatives in Journal of Neuro-Ophthalmology

Retrieved on: 
Thursday, May 19, 2022
Science, Biotechnology, Research, Pharmaceutical, Optical, Health, Genetics, Clinical Trials, Sequence, Work, Sex linkage, Parenting, Guilt, Literature, Participation, Systematic review, University, Moorfields Eye Hospital, National Eye Institute, Incidence, Natural history, Quality of life, Kawasaki, Marketing, Gray platelet syndrome, Research, Optogenetics, Mitochondrial disease, Severe cognitive impairment, Mitochondrion, Department, DNA, Company, Pain, Retina, Doctor of Philosophy, Partner, Patient, REALITY, Eye, Shoulder, Retinal ganglion cell, MTS, VF-14, Disability, MD, BioMed Central, Optic neuropathy, ISIN, Science, University of Cambridge, Neuro, Life, LHON, Union, National Eye, Leber's hereditary optic neuropathy, Mutation, Kawasaki disease, Diagnosis, Cataract, Pharmaceutical industry, Medical device, Medicine, Medical imaging, Phosphorus, Euronext

The study determined that the impact of LHON extends beyond vision-related activity limitations, while addressing its psychosocial impact.

Key Points: 
  • The study determined that the impact of LHON extends beyond vision-related activity limitations, while addressing its psychosocial impact.
  • Additionally, the study determined that despite residing in different countries, LHON patients and their relatives described similar experiences in the four areas of focus in this study.
  • The objective of this study was to comprehensively explore the impact of LHON on the lives of patients and their relatives at the time of diagnosis and now.
  • The Impact of Leber Hereditary Optic Neuropathy on the Quality of Life of Patients and Their Relatives: A Qualitative Study
    1.

Neurogene Announces New Development Program in Rett Syndrome Utilizing Novel EXACT Technology Platform

Retrieved on: 
Wednesday, May 18, 2022
Health, Genetics, General Health, Research, Science, Biotechnology, Construct, Phenotype, MECP2, Doctor of Philosophy, Sex linkage, Disability in Canada, Seizure, Rigidity, Survival, Incidence, Patient, Frasier, RSRT, Lists of diseases, Rett syndrome, Society, Mortality, Inc., University, Central nervous system disease, Research, Gene, Mouse, CNS, Neurodevelopmental disorder, Brain, Nervous system, Female, Center, Toxicity, Simons Foundation, Safety, Technology, Muscle contracture, Dementia, Mutation, Cell, Male, Speech, Tissue, CEO, Vaccine, Pharmaceutical industry, Rett, Neuroscience

Rett syndrome is a complex disorder not readily addressable by conventional gene therapy because the MECP2 gene is toxic if expressed at high levels.

Key Points: 
  • Rett syndrome is a complex disorder not readily addressable by conventional gene therapy because the MECP2 gene is toxic if expressed at high levels.
  • Neurogene is grateful for RSRTs support in advancing the scientific knowledge for the treatment of Rett syndrome, which ultimately enabled the early development of the EXACT technology.
  • Neurogene is also appreciative of the broader Rett syndrome patient community, including the International Rett Syndrome Foundation and Reverse Rett (UK).
  • We hope this novel approach will continue to advance in its development and ultimately provide a much-needed treatment option for families impacted by Rett syndrome.

ADMA Biologics Announces First Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, May 11, 2022
Wiskott–Aldrich syndrome, ADMA, Disease, Sex linkage, Infection, U.S. Securities and Exchange Commission, Antibody, PIDD, Plasma, Virus, IVIG, Adoption, Private Securities Litigation Reform Act, SEC, Collection, Physician, Income, Marketing, Food and Drug Administration, Respiratory syncytial virus, Growth, GLOBE, Company, General manager, Bacteria, Immunodeficiency, FDA, Hepatitis B virus, Lists of diseases, Protein, RSV, European Patent Office, Risk, ID, Nasdaq, Environment, Patient, Forward-looking statement, Immunity, 8-K, San Jose BioCenter, Morgan Stanley, Video game, Risk management, Pharmaceutical industry, Security (finance), Medical device, Vaccine, PI

RAMSEY, N.J. and BOCA RATON, Fla., May 11, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (“ADMA” or the “Company”), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced its first quarter 2022 financial results and provided a business update.  

Key Points: 
  • First Quarter 2022 Total Revenues Were Approximately $29.1 Million, an 81% Increase Over First Quarter 2021
    RAMSEY, N.J. and BOCA RATON, Fla., May 11, 2022 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA or the Company), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced its first quarter 2022 financial results and provided a business update.
  • We are very encouraged with our first quarter financial results, particularly when considering some of the non-recurring dynamics that occurred during the quarter, said Brian Lenz, ADMAs Chief Financial Officer, and General Manager, ADMA BioCenters.
  • First Quarter 2022 and Recent Achievements:
    Executed Financially: Achieved first quarter 2022 total revenues of $29.1 million, as compared to $16.0 million during the first quarter of 2021, an increase of $13.1 million, or approximately 81%.
  • 2022 & Long-Term Financial Guidance:
    2022 Financial Guidance: Enabled by the strong start to the year, ADMA increases 2022 total revenue guidance to $130 million or more, upwardly revised from $125 million.

Myrtelle Inc. Announces Expansion of Its Pipeline with a Novel Gene Therapy Program for Pelizaeus-Merzbacher Disease (PMD)

Retrieved on: 
Wednesday, May 11, 2022
Science, Other Science, Biotechnology, Research, Health, Genetics, Clinical Trials, Other Health, Architecture, Disorder, Hope, Sex linkage, Pfizer, Mutation, Brain, Inc., MicroRNA, CNS, Mirna, Death, Secondary research, Phenotype, Patient, Doctor of Philosophy, Stridor, White matter, Neurodegeneration, Gene silencing, PLP1, Human, Pelizaeus–Merzbacher disease, ASPA, Therapy, Hypotonia, Pharmacology, Disease, Science, Kaay Raav Tumhi, Intellectual property, Life, Nystagmus, Canavan disease, Central nervous system, Cell, Myrtelle Canavan, PMD, Pharmaceutical industry, Copper, Vaccine, Tight junction protein 1

This duplication results in excessive Plp1 accumulation that disrupts proper myelin formation and triggers extensive loss of oligodendrocytes in the CNS.

Key Points: 
  • This duplication results in excessive Plp1 accumulation that disrupts proper myelin formation and triggers extensive loss of oligodendrocytes in the CNS.
  • Oligodendrocytes are the cells in the brain responsible for producing myelin the insulating material that enables proper neuronal function.
  • Myrtelle has built enabling technologies and capabilities in gene therapy for disorders involving oligodendrocytes and myelin production.
  • Myrtelle has an exclusive worldwide licensing agreement with Pfizer for its lead program in Canavan disease.

Poxel Announces PXL770 Awarded FDA Fast Track Designation for X-linked Adrenoleukodystrophy

Retrieved on: 
Monday, April 11, 2022
Health, FDA, Genetics, Other Health, General Health, Pharmaceutical, Biotechnology, Risk, FTD, AMN, Conditional sentence, Metabolic disorder, TZD, AMPK, Incontinence, White matter, Medicine, ALD, Chronic inflammatory demyelinating polyneuropathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Haemophilia, NDA, VLCFA, Disease, ABCD1, Hematopoietic stem cell transplantation, Fatty liver disease, Peripheral nervous system, Adrenoleukodystrophy, Diagnosis, Peroxisome, Adenosine, POC, NASH, Sex linkage, Brain, Lists of diseases, Life, Metabolism, Fast Track, Forward-looking statement, Death, Gait, Growth, Euronext Paris, HSCT, Man, Accelerated approval (FDA), Review, Priority review, Weakness, Degenerative disease, FDA, Patient, Partnership, Spinal cord, Company, Cell, Woman, Food, Prevalence, Pharmaceutical industry, Medical device, Imeglimin, Euronext

Poxel CEO, Thomas Kuhn, commented: "Having PXL770 awarded Fast Track Designation by the FDA soon after our other promising product, PXL065, is a strong recognition of the potential of both our programs in adrenoleukodystrophy, a significant unmet medical need.

Key Points: 
  • Poxel CEO, Thomas Kuhn, commented: "Having PXL770 awarded Fast Track Designation by the FDA soon after our other promising product, PXL065, is a strong recognition of the potential of both our programs in adrenoleukodystrophy, a significant unmet medical need.
  • FTD provides Poxel with substantially enhanced access to FDA, including opportunities for face-to-face meetings and written consultations throughout the remaining development of PXL770.
  • Introduced under the FDA Modernization Act (1997), Fast Track Designation (FTD) may be awarded by the FDA to investigational drugs which treat a serious or life-threatening condition, and which fill an unmet medical need.
  • The FDA notes that 'the purpose of the Fast Track program is to get important new drugs to the patient earlier1.