The Pipeline Blog
In The Pipeline Blog, we aim to discuss a variety of medical and regulatory affairs topics.
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IMAAVY for the treatment of generalized myasthenia gravis
Author: Stacy Pitcairn Editor: Meghan Diefenbacher What is Myasthenia gravis? Generalized myasthenia gravis is a rare autoimmune neuromuscular disease characterized by weakness in voluntary muscles, including those used for movement, breathing, and swallowing.1 For most people, the first symptoms involve … Read more
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Lenacapavir: Paving the way for long-acting antiretroviral therapy (LA-ART) for treatment and prevention of human immunodeficiency virus (HIV) infections
Writer: Meghan Diefenbacher Editor: Kaitlin Kinney HIV-1 Global Public Health Burden Human immunodeficiency virus (HIV-1) remains a significant global public health burden. Since the HIV-1 pandemic began in the 1980s, it has claimed over 44.1 million lives, and, currently, 39.4 … Read more
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Lumipulse G pTau217/β-Amyloid 1-42 Plasma Ratio to Detect Alzheimer’s Disease
Author: Pari Dhayagude Editor: Grace Stroman Alzheimer’s disease (AD) remains one of the most challenging neurodegenerative disorders to diagnose in its early stages.1 Before the 2000s, a postmortem autopsy was the only definitive method for diagnosing AD. Since then, progress … Read more
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Jascayd (nerandomilast) and the Treatment of Idiopathic Pulmonary Fibrosis
Author: Sarah Sizer, Ph.D. Editor: Jessica Sprinkles This article will discuss Jascayd (nerandomilast), a phosphodiesterase 4B inhibitor developed by the pharmaceutical company Boehringer Ingelheim. In October 2025, the Food and Drug Administration (FDA) approved Jascayd for the treatment of idiopathic … Read more
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Investigations into the molecular etiology of hypermobile Ehlers-Danlos syndrome
Author: Tiffany Ko Editor: Karen Wang Overview of hypermobile Ehlers-Danlos Syndrome Hypermobile Ehlers-Danlos syndrome (hEDS) is one of the thirteen subtypes of Ehlers-Danlos syndrome (EDS), which are typically characterized by varying degrees of hypermobile joints, hyperextensible skin, and fragile tissues … Read more
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Aceclidine for Presbyopia: A New Horizon for Age-Related Blurry Vision?
Author: Stacy Pitcairn Editor: Kaitlin Kinney Overview As individuals age, many experience the onset of presbyopia. Presbyopia is a term that refers to the gradual loss of the eye’s ability to change its focus. Patients often report increasing difficulty concentrating … Read more
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Sevasemten for Becker Muscular Dystrophy: A Promising New Treatment Approach
Author – Kaitlin KinneyEditor – Grace Stroman For adults living with Becker muscular dystrophy (BMD), treatment options have been limited. While Duchenne muscular dystrophy (DMD) has seen multiple therapies approved in recent years, including gene therapies,1 exon-skipping drugs,2-5 and novel … Read more
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Adaptive Deep Brain Stimulation: A Personalized Approach to Parkinson’s Disease Management
Author- Pari Dhayagude Editor- Sarah Sizer, PhD Parkinson’s disease (PD) is the second most common neurodegenerative disease, affecting over 1 million people in the United States and over 10 million people worldwide.1 PD is a progressive neurological condition defined by … Read more
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Breaking the Block: Why Tenecteplase is a New Contender in Ischemic Stroke Therapy
Author: Colin McArdle Editor: Morgan McCullough Approximately every 40 seconds, someone will suffer from a stroke.1 As one of the top burdensome neurological disorders and the second leading cause of global deaths, roughly 94 million people worldwide are living with … Read more
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Crinecerfont for classic congenital adrenal hyperplasia
Author: Maya BluittEditor: Kéita Yokoyama The U.S. Food and Drug Administration recently approved a first-in-class medication for the treatment of classic congenital adrenal hyperplasia (CAH).1 Crenessity (crinecerfont), developed by Neurocrine Biosciences, acts to control adrenal androgens and revolutionizes treatment for … Read more