February 2024 – Page 3 – Patient Worthy

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

Post author:Jessica Lynn
Post published:February 8, 2024
Post category:Bile duct cancer Biliary tract cancer Cholangiocarcinoma

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.…

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Research Team Develops Technology That Could Make Gene Therapy Safer

Post author:Rose Duesterwald
Post published:February 7, 2024
Post category:Rare Disease

A Baylor College team of researchers headed by author and Associate Professor of Pathology, Liaising Yen, have spent over ten years working on a technology to effectively regulate gene expression.…

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

Post author:Jessica Lynn
Post published:February 7, 2024
Post category:Atopic dermatitis

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…

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HYQVIA and ENHANZE Co-Formulation Approved as Maintenance Therapy for CIDP by European Commission

Post author:Jessica Lynn
Post published:February 7, 2024
Post category:Chronic Inflammatory Demyelinating Polyneuropathy

In January 2024, the FDA approved Hyqvia (Immune Globulin Infusion 10% [Human]) as a maintenance therapy for adults living with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder. As…

source: pixabay.com

February is Turner Syndrome Awareness Month: Spreading Rare Disease Awareness

Post author:James Moore
Post published:February 7, 2024
Post category:Turner Syndrome

The month of February is recognized as Turner Syndrome Awareness Month, a time to spread awareness about this rare disease among the medical field and the general public. The Turner…

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Family Raises PKAN Awareness and Funds with Twins’ Story

Post author:Jessica Lynn
Post published:February 6, 2024
Post category:Pantothenate kinase-associated neurodegenerat Pantothenate Kinase-associated Neurodegeneration

The Binder family never expected that their 12-year-old twins, Grady and Jace, would be diagnosed with a rare genetic disorder. In fact, this particular disorder is so rare that it…

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FDA Approves Gammagard Liquid for CIDP

Post author:Jessica Lynn
Post published:February 6, 2024
Post category:Chronic Inflammatory Demyelinating Polyneuropathy

People with chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder, now have another treatment option: Gammagard Liquid (immune globulin infusion [human] 10% solution). Healio reports that the Food and…

source: shutterstock.com

The FDA Approves Agamree for Duchenne Muscular Dystrophy

Post author:Rose Duesterwald
Post published:February 6, 2024
Post category:Duchenne Muscular Dystrophy

The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone…

Fotocitizen / Pixabay

Rare Community Profiles: Dr. Pisit “Duke” Pitukcheewanont Discusses Phase 2 Results in LUM-201 for Pediatric Growth Hormone Deficiency

Post author:Jessica Lynn
Post published:February 6, 2024
Post category:Growth hormone deficiency Pediatric Growth Hormone Deficiency (GHD)

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.…

source: pixabay.com

Study of the Week: Contaminated Bedding Doubles Risk of C. Diff

Post author:James Moore
Post published:February 5, 2024
Post category:C. Diff

Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…

rawpixel / Pixabay

Patricia Brown Joins CureDuchenne as Senior Director of Engagement to Advance Mission of Finding Duchenne Muscular Dystrophy Cure

Post author:Jessica Lynn
Post published:February 5, 2024
Post category:Duchenne Muscular Dystrophy

For over 20 years, CureDuchenne, a Patient Worthy partner and global nonprofit, has moved steadfastly forward with a mission to find, and fund, a cure for individuals with Duchenne muscular…

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Sarepta is Pushing to Expand Duchenne Muscular Dystrophy Drug Label Despite Missed Endpoints

Post author:Rose Duesterwald
Post published:February 5, 2024
Post category:Duchenne Muscular Dystrophy

Sarepta Therapeutics’ treatment Elevidys (delandistrogene moxeparvovec-rokl) received conditional approval from the FDA in June 2023. The designation implies that if the drug is used in accordance with its label, it…

Rare Disease Day: Share Your Stripes with Patient Worthy!

Post author:James Moore
Post published:February 5, 2024
Post category:Rare Disease

Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…

1662222 / Pixabay

Contaminated Bed Use in Hospitals Significantly Increases Risk of C. Diff Infection

Post author:Jessica Lynn
Post published:February 2, 2024
Post category:C. Diff

Researchers have made an important discovery which indicates that individuals are increasingly likely to contact Clostridioides difficile (C. diff) when occupying a hospital room or bed previously used by a…

Photo by Wengang Zhai on Unsplash

ASCO GI 2024: Oral Antroquinonol Improved Overall Survival Rates in Metastatic Pancreatic Cancer

Post author:Jessica Lynn
Post published:February 2, 2024
Post category:Pancreatic Cancer

The American Society of Clinical Oncology (ASCO) held its ASCO Gastrointestinal Cancers Symposium from January 18-20, 2024. During the Symposium, doctors, researchers, scientists, and other stakeholders discussed innovative science and…

Knowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities

Post author:Jessica Lynn
Post published:February 2, 2024
Post category:Duchenne Muscular Dystrophy

CureDuchenne, a global leader in research and patient care for Duchenne muscular dystrophy (DMD), has been working to find and fund a cure for DMD for more than two decades.…

source: shutterstock

Rare Classroom: Metastatic Urothelial Cancer

Post author:James Moore
Post published:February 2, 2024
Post category:Bladder cancer Urothelial Carcinoma

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

Controversial Alzheimer’s drug exits the stage

Post author:Rose Duesterwald
Post published:February 1, 2024
Post category:Rare Disease

Controversial Alzheimer's drug comes off the market (axios.com)

source: shutterstock.com

Visual Snow Syndrome 101

Post author:Patient Worthy Contributor
Post published:February 1, 2024
Post category:Visual snow syndrome

Written by Sierra Domb What Is Visual Snow Syndrome (VSS)? Visual snow syndrome (VSS) is a neurological disorder that can impact an individual’s vision, hearing, cognition, sensory processing, and quality…

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Study: Private Equity-Owned Hospitals Less Safe for Patients

Post author:James Moore
Post published:February 1, 2024
Post category:Rare Disease

According to an article published in Healthcare Dive, a recent study published in the academic journal JAMA indicated that patients receiving treatment in hospitals owned by private equity firms were at an…

Rare Disease Day: Share Your Stripes with Patient Worthy!

Post author:James Moore
Post published:February 1, 2024
Post category:Rare Disease

Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…

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

Research Team Develops Technology That Could Make Gene Therapy Safer

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

HYQVIA and ENHANZE Co-Formulation Approved as Maintenance Therapy for CIDP by European Commission

February is Turner Syndrome Awareness Month: Spreading Rare Disease Awareness

Family Raises PKAN Awareness and Funds with Twins’ Story

FDA Approves Gammagard Liquid for CIDP

The FDA Approves Agamree for Duchenne Muscular Dystrophy

Rare Community Profiles: Dr. Pisit “Duke” Pitukcheewanont Discusses Phase 2 Results in LUM-201 for Pediatric Growth Hormone Deficiency

Study of the Week: Contaminated Bedding Doubles Risk of C. Diff

Patricia Brown Joins CureDuchenne as Senior Director of Engagement to Advance Mission of Finding Duchenne Muscular Dystrophy Cure

Sarepta is Pushing to Expand Duchenne Muscular Dystrophy Drug Label Despite Missed Endpoints

Rare Disease Day: Share Your Stripes with Patient Worthy!

Contaminated Bed Use in Hospitals Significantly Increases Risk of C. Diff Infection

ASCO GI 2024: Oral Antroquinonol Improved Overall Survival Rates in Metastatic Pancreatic Cancer

Knowledge is Power: CureDuchenne Cares Educational Events Aim to Equip Families and Caregivers in the Becker and Duchenne Muscular Dystrophy Communities

Rare Classroom: Metastatic Urothelial Cancer

Controversial Alzheimer’s drug exits the stage

Visual Snow Syndrome 101

Study: Private Equity-Owned Hospitals Less Safe for Patients

Rare Disease Day: Share Your Stripes with Patient Worthy!

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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