Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

    Experimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study
    source: shutterstock.com

    Experimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study

    Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…

    Continue Reading Experimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study
    Drafting a Dream Team: Celebrating Breakthroughs from the February 2024 Uplifting Athletes Young Investigator Draft
    Photo Courtesy of Uplifting Athletes

    Drafting a Dream Team: Celebrating Breakthroughs from the February 2024 Uplifting Athletes Young Investigator Draft

    Having a rare disease can be challenging and isolating. It's often difficult to describe the experience to someone who hasn't gone through it. Mobilizing funds for support, resources, and research…

    Continue Reading Drafting a Dream Team: Celebrating Breakthroughs from the February 2024 Uplifting Athletes Young Investigator Draft
    Could Simulated Sunshine Improve Psycho-Behavioral Symptoms in Alzheimer’s Disease?
    Source: pixabay.com

    Could Simulated Sunshine Improve Psycho-Behavioral Symptoms in Alzheimer’s Disease?

    Alzheimer’s disease is a progressive neurodegenerative disease and the most common form of dementia. People with Alzheimer’s disease may have difficulty remembering information, problems with learning, sleep disruptions, poor judgment,…

    Continue Reading Could Simulated Sunshine Improve Psycho-Behavioral Symptoms in Alzheimer’s Disease?
    Zachary Thomas Newborn Screening Act Could Introduce MPS I to Alabama’s Newborn Screening Panel
    source: shutterstock.com

    Zachary Thomas Newborn Screening Act Could Introduce MPS I to Alabama’s Newborn Screening Panel

    Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born.  Newborn screening is a public health initiative that tests newborn babies for…

    Continue Reading Zachary Thomas Newborn Screening Act Could Introduce MPS I to Alabama’s Newborn Screening Panel
    Rare Community Profiles: Breaking Barriers: How Nicole and Emma Offer Hope Amidst a Dravet Syndrome Diagnosis
    source: pixabay.com

    Rare Community Profiles: Breaking Barriers: How Nicole and Emma Offer Hope Amidst a Dravet Syndrome Diagnosis

      Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

    Continue Reading Rare Community Profiles: Breaking Barriers: How Nicole and Emma Offer Hope Amidst a Dravet Syndrome Diagnosis
    Auditory Sensory Memory System Decline: A Potential Neuromarker for Juvenile-Onset Batten Disease
    geralt / Pixabay

    Auditory Sensory Memory System Decline: A Potential Neuromarker for Juvenile-Onset Batten Disease

    In total, there are thirteen subtypes of Batten disease. Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a rare and inherited neurodegenerative disorder that causes visual impairment, behavioral…

    Continue Reading Auditory Sensory Memory System Decline: A Potential Neuromarker for Juvenile-Onset Batten Disease
    ClimbFARACure: How Summiting Aconcagua Raised Friedreich’s Ataxia Awareness
    source: pixabay.com

    ClimbFARACure: How Summiting Aconcagua Raised Friedreich’s Ataxia Awareness

    It was never just about reaching the summit of Aconcagua, the highest mountain in both the Western and Southern hemispheres. For Scott Osleeb, scaling the heights of this remarkable peak…

    Continue Reading ClimbFARACure: How Summiting Aconcagua Raised Friedreich’s Ataxia Awareness
    Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction
    source: shutterstock.com

    Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction

    What role do brain alterations play in the onset and progression of spinocerebellar ataxia type 6 (SCA6)? While previous studies highlighted cerebellar changes in individuals with SCA6, the underlying mechanisms…

    Continue Reading Navigating the Neurological Maze: McGill’s Breakthrough Study Explores SCA6 and Endosomal Dysfunction
    Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)
    JerzyGorecki / Pixabay

    Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)

    Developing novel therapeutics for rare diseases is increasingly important. Understanding the unmet needs of rare disease communities and providing effective, accessible therapies can significantly improve the lives of those affected.…

    Continue Reading Ruxoprubart Granted Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Florida State University Harnesses Interdisciplinary Collaboration Through New Institute for Pediatric Rare Diseases
    source: pixabay.com

    Florida State University Harnesses Interdisciplinary Collaboration Through New Institute for Pediatric Rare Diseases

    When it comes to rare disease, rare isn't actually that rare. More than 10,000 rare diseases have been identified. Rare diseases affect more than 350 million people across the globe…

    Continue Reading Florida State University Harnesses Interdisciplinary Collaboration Through New Institute for Pediatric Rare Diseases
    Dupixent Label Expands to Include Children Ages 1-11 with Eosinophilic Esophagitis
    source: pixabay.com

    Dupixent Label Expands to Include Children Ages 1-11 with Eosinophilic Esophagitis

    In late January 2024, the U.S. Food and Drug Administration approved a label expansion for Dupixent (dupilumab). Dupixent, a monoclonal antibody targeting IL-4 and IL-13, is currently used to treat…

    Continue Reading Dupixent Label Expands to Include Children Ages 1-11 with Eosinophilic Esophagitis
    Rare Community Profiles: Unraveling the Genetic Threads: Erin’s Quest to Test for ALSP
    Fotocitizen / Pixabay

    Rare Community Profiles: Unraveling the Genetic Threads: Erin’s Quest to Test for ALSP

      Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

    Continue Reading Rare Community Profiles: Unraveling the Genetic Threads: Erin’s Quest to Test for ALSP
    Four-Year-Old Boy Tackles Sturge-Weber Syndrome Through Presymptomatic Treatment
    https://pixabay.com/en/boy-people-silhouette-bokeh-blur-1209000/

    Four-Year-Old Boy Tackles Sturge-Weber Syndrome Through Presymptomatic Treatment

    At four and a half years old, Tucker Lewis is thriving. He loves playing with his friends, riding his bike outside, and watching the Atlanta Braves (He's even going to…

    Continue Reading Four-Year-Old Boy Tackles Sturge-Weber Syndrome Through Presymptomatic Treatment
    STUDY: Palliative Telecare Conferred Quality-of-Life Improvements in COPD
    Source: https://pixabay.com/en/upper-body-lung-copd-disease-944557/

    STUDY: Palliative Telecare Conferred Quality-of-Life Improvements in COPD

    Treatments and management plans exist for chronic obstructive pulmonary disease (COPD), heart failure, and interstitial lung disease (ILD). Yet despite these treatments, many people with these conditions still experience chronic…

    Continue Reading STUDY: Palliative Telecare Conferred Quality-of-Life Improvements in COPD

    Pre-Clinical Safety Data Available on DA-1241 Combination for Metabolic Dysfunction-Associated Steatohepatitis (MASH)

    Metabolic dysfunction-associated steatohepatitis (MASH) unfortunately has no treatments to reverse the disease or its damage. MASH can be managed through weight loss, regular exercise, and blood sugar management. But identifying…

    Continue Reading Pre-Clinical Safety Data Available on DA-1241 Combination for Metabolic Dysfunction-Associated Steatohepatitis (MASH)
    ASCO GI Symposium: Fostrox-Lenvima Combo Treatment Show Clinical Benefit in Treating HCC
    source: shutterstock.com

    ASCO GI Symposium: Fostrox-Lenvima Combo Treatment Show Clinical Benefit in Treating HCC

    Despite novel treatment advances, hepatocellular carcinoma (HCC) remains difficult to treat. This aggressive cancer is often not found until later stages and not all people with HCC can utilize available…

    Continue Reading ASCO GI Symposium: Fostrox-Lenvima Combo Treatment Show Clinical Benefit in Treating HCC
    Rare Community Profiles: From Stage 4 Cholangiocarcinoma to Cancer-Free in 12 Months: How Hepatic Artery Infusion (HAI) Changed Rick’s Trajectory
    Fotocitizen / Pixabay

    Rare Community Profiles: From Stage 4 Cholangiocarcinoma to Cancer-Free in 12 Months: How Hepatic Artery Infusion (HAI) Changed Rick’s Trajectory

      Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

    Continue Reading Rare Community Profiles: From Stage 4 Cholangiocarcinoma to Cancer-Free in 12 Months: How Hepatic Artery Infusion (HAI) Changed Rick’s Trajectory
    Dupixent Label Update Reflects Safety and Efficacy Results for Moderate-to-Severe Atopic Dermatitis with Severe Hand/Foot Involvement
    source: shutterstock.com

    Dupixent Label Update Reflects Safety and Efficacy Results for Moderate-to-Severe Atopic Dermatitis with Severe Hand/Foot Involvement

    According to reporting from Healio Psoriatic Disease, the Food and Drug Administration (FDA) recently updated the label for Dupixent (dupilumab) in the indication of moderate-to-severe atopic dermatitis with moderate-to-severe hand…

    Continue Reading Dupixent Label Update Reflects Safety and Efficacy Results for Moderate-to-Severe Atopic Dermatitis with Severe Hand/Foot Involvement