BIOL391 Research - Philosophy of Experimentation (Ch1-2) PDF

9/3/24 Chapter 1 Philosophy of Glass, D.J. (2014) Experimental Design for Experimentation Biologists. 2nd ed., Cold Spring Harbor Ch....

9/3/24 Chapter 1 Philosophy of Glass, D.J. (2014) Experimental Design for Experimentation Biologists. 2nd ed., Cold Spring Harbor Ch. 1-5, Glass (2014) Laboratory Press, Cold Spring Harbor, New York. BIOL 391 Why You Need to Read a Little Philosophy First. Intro to Biological Research The Philosophy of Science Governs the Burman University, Fall 2024 Practice of Science. 1 2 Epistemology Epistemology Historically has dealt with the following Epistemology is the study of fundamental questions: knowledge – it limits and validity. – What is knowledge, and what do we mean when we say that we know something? – derived from the Greek words epistéme – What is the source of knowledge, and how (“knowledge”) and logos (“study of”). do we know if it is reliable? – What is the scope of knowledge, and what are its limitations? 3 4 Scientific “Process” Practical Scientific Epistemology The way in which scientists must approach and conduct their experiments. How can we produce and interpret a data set that can be reproduced by others? The essential training as to how science should be performed. How can we come to a conclusion that can be validated by its predictive power? 5 6 1  9/3/24 Search All Register Sign In Browse all medications: A B C D E F G H I J K L M N O P Q R S T U V W X Y Z 0-9 Advanced Search DRUGS A-Z PILL IDENTIFIER INTERACTION CHECKER NEWS & ALERTS PRO EDITION MORE ADVERTISEMENT PRICE DROP PRICE DROP PRICE DROP PRICE DROP Chapter 2 Defining Scientific Research ADVERTISEMENT Home Articles Print 10 of the Most Expensive Drugs in the U.S. Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on April 1, 2024. PRICE DROP 1. Lenmeldy Cost: $4.25 million per one time treatment Manufacturer: Orchard Therapeutics PRICE DROP Use: Metachromatic leukodystrophy (MLD) FDA Approval Date: March 18, 2024 Lenmeldy (atidarsagene autotemcel) is an autologous hematopoietic stem cell (HSC) gene therapy for the treatment of children with metachromatic leukodystrophy (MLD). Lenmeldy is the first treatment for MLD PRICE DROP and has the potential to restore enzymatic function to stop or slow this fatal disease with a single treatment. Lenmeldy will be given at Qualified Treatment Centers in some U.S. states. The wholesale acquisition cost (WAC) of Lenmeldy will be US$4.25 million according to Orchard Therapeutics, making it currently the most expensive drug in the U.S. Officials at Orchard cite the value of 7 8 Temu on sale therapy to patients and their families, possible caregiver wage loss, and the potential to lower healthcare Temu utilization. MLD is a rare and potentially fatal genetic disease of the metabolic system caused by a mutation in the Subscribe to our newsletters arylsulfatase-A (ARSA) gene. This mutation causes accumulation of sulfatides (a type of fat) that destroy Monthly newsletter the protective layer of the nerves called the myelin sheath. Patients eventually lose the ability to move, Daily MedNews talk, swallow, eat and see. Mortality is estimated at 50% at 5 years for late infantile disease and 44% at 10 FDA safety alerts years for juvenile patients. I accept the Terms and Privacy Policy Lenmeldy works by inserting one or more functional copies of the human ARSA gene into the genome of a patient’s own hematopoietic stem cells (HSCs), outside the body, using a lentiviral vector. Cells are then Email address Subscribe MLD infused back into the patient where they can migrate across the blood-brain barrier into the central nervous system and express the functional enzyme. ADVERTISEMENT Metachromatic leukodystrophy is a rare and Open-label studies looked at data from 37 pediatric patients with early-onset MLD with 12 years of follow- severe genetic condition that affects the brain and up (median 6.8 years). All children with pre-symptomatic late infantile MLD who were treated with Lenmeldy were alive at 6 years of age, compared to 58% in the natural history group. At 5 years of age, nervous system. 71% of treated children were able to walk without assistance. Metachromatic leukodystrophy is caused by a faulty The most common adverse reactions (≥10%) include fever and low white blood cell count, mouth sores, PRICE DROP respiratory infections, rash, medical line infections, viral infections, fever, gastrointestinal infections and gene, an enzyme called arylsulfatase A (ARSA). enlarged liver. That lack of ARSA causes fatty substances called ADVERTISEMENT Search For sulfatides to build up in the brain and nerves, 1 5 MOST EXPENSIVE MEDICATIONS › 3 BEST ASTHMA MEDICATION › leading to problems with movement and thinking 2 TOP 5 MEDICARE ADVANTAGE PLANS › 4 OZEMPIC PRICE › abilities, severe spasticity, seizures, and patients gradually losing the ability to move, talk, swallow, 2. Hemgenix PRICE DROP PRICE DROP eat, and see. Cost: $3.5 million per one-time dose Lenmeldy therapy replaces the faulty gene in the MR images of three pa0ents with MLD. Manufacturer: CSL Behring YU, C. (2017). LYSOSOMAL STORAGE DISORDERS: SPHINGOLIPIDOSIS, CHAPTER 10. stem cells. Use: Hemophilia BIOMARKERS IN INBORNB ERRORS OF METABOLISM. AMSTERDAM: ELSEVIER, 211-33. Temu on sale FDA Approval Date: November 22, 2022 Temu 9 10 Hemgenix (etranacogene dezaparvovec-drlb) is approved to treat hemophilia B, a rare, lifelong bleeding disorder. Given as a single, one-time intravenous (IV) infusion, it's list price of $3.5 million makes it the most expensive drug in the U.S. It allows people living with hemophilia B to produce their own factor IX (a blood clotting factor) and can lower the risk of bleeding with a single dose. Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps to form blood clots. People with the condition are particularly vulnerable to bleeding in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Typically, patients with hemophilia B have to adhere to strict, expensive, lifelong infusion schedules of factor IX. ADDYI (flibanserin) Hemgenix is an adeno-associated virus vector-based gene therapy. It works by supplying a noninfectious viral vector (AAV5) to carry genetic DNA instructions to the liver, where factor IX proteins are then generated. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. In the HOPE-B clinical trial, Hemgenix reduced the rate of annual bleeds and 94% of patients discontinued factor IX infusions. Seven to 18 months after Hemgenix, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54%. The most commonly reported side effects (incidence ≥5%) with this gene therapy were liver enzyme elevations, headache, elevated blood enzymes, flu-like symptoms, infusion-related reactions, fatigue, nausea and feeling unwell. 3. Elevidys Cost: $3.2 million per one-time dose Manufacturer: Sarepta Therapeutics Use: Duchenne Muscular Dystrophy (DMD) ADVERTISEMENT FDA Approval Date: June 22, 2023 Elevidys (delandistrogene moxeparvovec-rokl) is approved for the treatment of children aged 4 through 5 years with Duchenne Muscular Dystrophy (DMD) with a confirmed mutation in the DMD gene. It's given as a one-time, single-dose intravenous (IV) infusion with a $3.2 million price tag. Patients selected for treatment have anti-AAVrh74 total binding antibody titers