Medium Chain Acyl CoA Dehydrogenase Deficiency

DUBLIN, Jan. 3, 2023 /PRNewswire/ -- The "Newborn Screening Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering. The global newborn screening market size reached US$ 880.2 Million in 2021. Looking forward, the publisher expects the market to reach US$ 1,371.7 Million by 2027, exhibiting a CAGR of 7.67% during 2021-2027. Companies Mentioned Agilent Technologies Inc. Baebies Inc. Bio-RAD Laboratories Inc. Chromsystems Instruments & Chemicals GmbH Danaher Corporation Masimo Corporation Medtronic plc Natus Medical Incorporated Perkinelmer Inc. RECIPE Chemicals + Instruments GmbH Thermo Fisher Scientific Inc. Trivitron Healthcare Waters Corporation Keeping in mind the uncertainties of COVID-19, we are continuously tracking and evaluating the direct as well as the indirect influence of the pandemic. These insights are included in the report as a major market contributor. Newborn screening is a public healthcare initiative focusing on infant screening, parental education, appropriate follow-up, diagnostic testing, disease management, and continued evaluation. It targets diseases that can cause significant morbidity, mortality, and intellectual disability (ID). Some of these diseases included in the newborn screening tests are galactosemia, phenylketonuria (PKU), homocystinuria, hearing loss, sickle cell disease (SCD), biotinidase deficiency, maple syrup urine, congenital adrenal hyperplasia, and medium-chain acyl-CoA dehydrogenase (MCAD) deficiency. Nowadays, public healthcare organizations across the globe are adopting new testing techniques, such as tandem mass spectrometry (MS/MS), to detect disorders associated with protein and fatty acid metabolism using blood samples. The rising prevalence of congenital conditions among infants, along with increasing concerns among parents about the health of children, represents one of the significant factors influencing the market. Newborn screening relies on a specific set of laboratory evaluations and point-of-care examinations for early detection, diagnosis, and intervention of disorders. These disorders are genetic, metabolic, blood, or hormone-related, which are not immediately apparent after delivery and require practical intervention. This, coupled with the rising awareness about early diagnosis and improving diagnostic modalities, is propelling the growth of the market. Moreover, with the development of robust immunoassays and molecular diagnostics, disorders like primary hypothyroidism, cystic fibrosis (CF), and congenital toxoplasmosis are being targeted in several newborn screening tests. Apart from this, governing agencies of numerous countries are planning to mandate newborn screening programs due to increasing child morbidity and mortality, which is also contributing to market growth. Furthermore, the leading players are engaged in building strategic partnerships to ensure product sustainability, which is anticipated to create a positive outlook for the market. Key Questions Answered in This Report: How has the global newborn screening market performed so far and how will it perform in the coming years? What has been the impact of COVID-19 on the global newborn screening market? What are the key regional markets? What is the breakup of the market based on the product? What is the breakup of the market based on the technology? What is the breakup of the market based on the test type? What are the various stages in the value chain of the industry? What are the key driving factors and challenges in the industry? What is the structure of the global newborn screening market and who are the key players? What is the degree of competition in the industry? Key Topics Covered: 1 Preface 2 Scope and Methodology 3 Executive Summary 4 Introduction 4.1 Overview 4.2 Key Industry Trends 5 Global Newborn Screening Market 5.1 Market Overview 5.2 Market Performance 5.3 Impact of COVID-19 5.4 Market Forecast 6 Market Breakup by Product 6.1 Instruments 6.1.1 Market Trends 6.1.2 Market Forecast 6.2 Reagents 6.2.1 Market Trends 6.2.2 Market Forecast 7 Market Breakup by Technology 7.1 Tandem Mass Spectrometry 7.1.1 Market Trends 7.1.2 Market Forecast 7.2 Pulse Oximetry 7.2.1 Market Trends 7.2.2 Market Forecast 7.3 Enzyme Based Assay 7.3.1 Market Trends 7.3.2 Market Forecast 7.4 DNA Assay 7.4.1 Market Trends 7.4.2 Market Forecast 7.5 Electrophoresis 7.5.1 Market Trends 7.5.2 Market Forecast 7.6 Others 7.6.1 Market Trends 7.6.2 Market Forecast 8 Market Breakup by Test Type 8.1 Dry Blood Spot Test 8.1.1 Market Trends 8.1.2 Market Forecast 8.2 CCHD 8.2.1 Market Trends 8.2.2 Market Forecast 8.3 Hearing Screen 8.3.1 Market Trends 8.3.2 Market Forecast 9 Market Breakup by Region 10 SWOT Analysis 11 Value Chain Analysis 12 Porters Five Forces Analysis 13 Price Analysis 14 Competitive Landscape 14.1 Market Structure 14.2 Key Players 14.3 Profiles of Key Players For more information about this report visit Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

TORONTO--(BUSINESS WIRE)-- FSD Pharma Inc. (NASDAQ: HUGE) (CSE: HUGE) (FRA: 0K9A) (“FSD Pharma” or the “Company”), a biopharmaceutical company dedicated to building a portfolio of innovative assets and biotech solutions to address ailments affecting millions worldwide, today announces recruiting is underway for the Company’s Phase 2 clinical trial of FSD201 for the treatment of chronic pain associated with idiopathic MCAS (MCAD) at two clinical sites in the USA, and a Canadian site to be ready to recruit soon. The trial and its status are available on ClinicalTrials.gov (Identifier: NCT05652907). FSD201 is a proprietary anti-inflammatory compound with the potential to address a wide range of inflammatory diseases and associated conditions. FSD201 successfully completed a Phase 1 safety and tolerability trial with topline results. The multi-center, randomized, double-blind, placebo controlled parallel group study will enroll 60 idiopathic MCAS patients. Per the protocol, patients will receive either 600-milligram tablets of FSD201 or placebo twice daily for 56 consecutive days. The primary outcome is a 30% decrease from baseline to day 28 in the average daily pain intensity. The trial will also evaluate many secondary outcomes. “FSD201 is a unique proprietary formulation of palmitoyl ethanolamide, and already received the new molecular entity (NME) designation for a potential 505(b)(1) application to the US FDA. This is attractive because of the potential exclusive regulatory access to the US market. Our clinical team put in a lot of thought into exploring unmet needs in inflammatory disorders and associated conditions. We are excited to evaluate the efficacy and safety of FSD201 in the treatment of chronic widespread musculoskeletal nociplastic pain associated with idiopathic MCAS, a disease that has challenged caregivers and researchers since the first case was diagnosed in 2007, and potentially millions of patients have a significantly reduced quality of life,” said Dr. Lakshmi P. Kotra, Head of FSD Pharma’s FSD Biosciences and CEO of Lucid Psycheceuticals. “Owing to the complexity of the disease, the therapeutic market is grossly underserved, presenting us with an opportunity as a pioneer in the space to provide a novel therapeutic option to scores of MCAS patients desperately in need of safe alternatives to the current standard of care. We feel we are in a position with only a handful of companies conducting clinical trials for this indication.” Mast cells are the first responders of the innate and adaptive immune systems, responding to endogenous factors to play an important role in anaphylaxis and tissue healing. MCAS refers to a group of disorders characterized by multisystem symptoms resulting from the accumulation of altered mast cells and/or abnormal mast cell mediator release, causing repeated anaphylactic symptoms/episodes and trapping a patient in a cycle of neurogenic pain and inflammation. Chronic widespread musculoskeletal nociplastic pain, arising from neurogenic inflammation, is associated with MCAS. Symptoms of MCAS can start at any age, but usually begin in adulthood. Due to the ubiquitous nature of mast cells throughout human body tissue, MCAS has the potential to affect every organ system, frequently without showing abnormalities in routine testing and patients suffer from chronic and systemic pain. Up to 30% of the general population can be affected by disorders related to mast cell activation (atopic disorders). Conversely, mastocytosis and MMAS (monoclonal mast cell activation syndrome) are typically considered rare diseases, affecting 1 in 10,000-20,000 subjects.1 The cause of MCAS is unknown and there is no cure. 1 (17)31025-4/pdf FSD also announces the grant of stock options to directors and officers of the Company to purchase up to an aggregate of 2,000,000 Class B subordinate voting shares in accordance with the Company’s stock option plan and subject to vesting terms. The options are exercisable at a price of C$1.30 per share and will expire five years from the date of grant. Certain Independent directors were granted 400,000 PSU’s. About FSD Pharma FSD Pharma Inc. is a biotechnology company with three drug candidates in different stages of development. FSD BioSciences, Inc., a wholly owned subsidiary, is focused on pharmaceutical research and development of its lead compound, FSD201, a proprietary ultra-micronized PEA formulation, for the treatment of inflammatory diseases. Lucid Psycheceuticals Inc., a wholly owned subsidiary, is focused on the research and development of its lead compounds, Lucid-Psych and Lucid-MS. Lucid-Psych is a molecular compound identified for the potential treatment of mental health disorders, and expanding this category, the Company is investigating other products addressing acute medical needs due to the abuse of drugs such as alcohol. Lucid-MS is a molecular compound identified for the potential treatment of neurodegenerative disorders. Forward Looking Information This press release contains forward-looking statements and forward-looking information (collectively, "forward-looking statements") within the meaning of applicable securities laws. Any statements that are contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements are often identified by terms such as “plans”, “expects”, “expected”, “scheduled”, “estimates”, “intends”, “anticipates”, “hopes”, “planned” or “believes”, or variations of such words and phrases, or states that certain actions, events or results “may”, “could”, “would”, “might”, “potentially” or “will” be taken, occur or be achieved. More particularly, and without limitation, this press release contains forward-looking statements contained in this press release include statements concerning the future of FSD Pharma Inc. and are based on certain assumptions that FSD Pharma has made in respect thereof as of the date of this press release. FSD Pharma cannot give any assurance that such forward-looking statements will prove to have been correct. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. The Company cautions that although it believes the expectations and material factors and assumptions reflected in these forward-looking statements are reasonable as of the date hereof, there can be no assurance that these expectations, factors and assumptions will prove to be correct and these risks and uncertainties give rise to the possibility that actual results may differ materially from the expectations set out in the forward-looking statements. These forward-looking statements are not guarantees of future performance and are subject to a number of known and unknown risks and uncertainties including, but not limited to: the fact that the drug development efforts of both Lucid and FSD BioSciences are at a very early stage; the fact that preclinical drug development is uncertain, and the drug product candidates of Lucid and FSD BioSciences may never advance to clinical trials; the fact that results of preclinical studies and early-stage clinical trials may not be predictive of the results of later stage clinical trials; the uncertain outcome, cost, and timing of product development activities, preclinical studies and clinical trials of Lucid and FSD BioSciences; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; the potential inability to obtain or maintain regulatory approval of the drug product candidates of Lucid and FSD BioSciences; the introduction of competing drugs that are safer, more effective or less expensive than, or otherwise superior to, the drug product candidates of Lucid and FSD BioSciences; the initiation, conduct, and completion of preclinical studies and clinical trials may be delayed, adversely affected, or impacted by COVID-19 related issues; the potential inability to obtain adequate financing; the potential inability to obtain or maintain intellectual property protection for the drug product candidates of Lucid and FSD BioSciences; and other risks. Accordingly, readers should not place undue reliance on the forward-looking statements contained in this press release, which speak only as of the date of this press release. Further information regarding factors that may cause actual results to differ materially are included in the Company’s annual and other reports filed from time to time with the Canadian Securities Administrators on SEDAR ( ) and with the U.S. Securities and Exchange Commission on EDGAR ( ), including the Company’s Annual Report on Form 20-F for the fiscal year ended December 31, 2021, under the heading “Risk Factors.” This list of risk factors should not be construed as exhaustive. Readers are cautioned that events or circumstances could cause results to differ materially from those predicted, forecasted or projected. The forward-looking statements contained in this document speak only as of the date of this document. FSD Pharma does not undertake any obligation to publicly update or revise any forward-looking statements or information contained herein, except as required by applicable laws. The forward-looking statements contained in this document are expressly qualified by this cautionary statement. Neither the Canadian Securities Exchange nor its regulation services provider accept responsibility for the adequacy or accuracy of this release.

GRAND RAPIDS, Mich., Sept. 15, 2021 /PRNewswire/ -- NxGen MDX is taking an innovative approach to genetic carrier screening panel design by creating the Early Advantage Panel. This panel is the first of its size to fully incorporate screening recommendations from the U.S. Department of Health and Human Services (HHS). By including conditions recommended by HHS for newborn screening (NBS), we are reiterating our focus on improving pregnancy and newborn health outcomes for families everywhere. September is Newborn Screening Awareness Month; each year, an estimated 4 million babies are reached by Newborn Screening (NBS) programs. While NBS is one of the most successful healthcare programs in the nation, it does have some limitations. The number of conditions screened is inconsistent from state to state with only 10 states screening for all HHS recommended conditions. Also, factors like birth weight, gestational age, ethnicity, and time of sample collection can lead to higher failure rates1,2. In contrast, NxGen's carrier screening is not dependent on geographical location, timing, or any characteristics of the infant, which allows for more equitable care. Symptoms of some genetic conditions can occur before NBS results are available, as many of the conditions recommended for universal screening can affect a baby's health in the first hours or days of life. For example, up to 24% of babies with medium-chain acyl-CoA dehydrogenase deficiency (MCADD) will show symptoms before NBS results are back3,4. Screening identifies at-risk infants and allows parents and doctors to implement appropriate intervention before an acute episode can take place. "Carrier screening can give parents time to prepare for these genetic conditions before their baby is born. NxGen designed this panel to provide early, accurate results that can help enable expedited diagnostic testing to inform treatment options, prevent long-lasting health issues, and potentially save a baby's life," said Jacqueline Peacock, PhD, Director of Laboratory Operations at NxGen. The Early Advantage Panel complements the NBS program by identifying at-risk couples before or during pregnancy, providing parents with a superior test, better reproductive options, and more timely information to manage their pregnancy and childbirth than relying on NBS alone. "NxGen follows a philosophy called NatalCare for all our products. NatalCare focuses on improving pregnancy and newborn outcomes. We believe that giving patients and providers vital information early on will allow them to make better healthcare decisions," says NxGen CEO, Alan Mack. Our NatalCare philosophy and commitment to best-in-class carrier screening provide parents with actionable information they can use to address genetic health issues their child may face immediately after birth with the potential to save lives. That is the early advantage. About NxGen MDx NxGen MDx, LLC is a leading healthcare company delivering comprehensive genetic testing. NxGen MDx's history of next-generation sequencing combined with advanced technology allows us to provide accessible, high-quality testing options to families as they plan for the future. 1. Slaughter, J.L., Meinzen-Derr, J., Rose, S.R., Leslie, N.D., Chandrasekar, R., Linard, S.M., and Akinbi, H.T. (2010). The Effects of Gestational Age and Birth Weight on False-Positive Newborn-Screening Rates. Pediatrics 126, 910–916. 2. Peng, G., Tang, Y., Cowan, T.M., Zhao, H., and Scharfe, C. (2021). Timing of Newborn Blood Collection Alters Metabolic Disease Screening Performance. Front. Pediatr. 8, 623184. 3. Ahrens-Nicklas RC, Pyle LC, Ficicioglu C. Morbidity and mortality among exclusively breastfed neonates with medium-chain acyl-CoA dehydrogenase deficiency. Genet Med. 2016 Dec;18(12):1315-1319. 4. Hsu HW. et al. Spectrum of medium-chain acyl-CoA dehydrogenase deficiency detected by newborn screening. Pediatrics. 2008 May;121(5):e1108-14. doi: 10.1542/ peds.2007-1993. PMID: 18450854.