2024 – Page 13 – Patient Worthy

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Rare Community Profiles: A Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa

Post author:Jessica Lynn
Post published:March 27, 2024
Post category:Lung Cancer Rare Disease

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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Positive Data Suggests NT-0796 Could Benefit People with Parkinson’s Disease

Post author:Jessica Lynn
Post published:March 26, 2024
Post category:Parkinson's Disease

Parkinson's disease is a progressive neurodegenerative disease. As neurons in the brain degenerate and die, those affected experience symptoms such as tremors or shaking in the hands, slowed movement, muscle…

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CABA-201 for Systemic Sclerosis Earns Orphan Drug Designation

Post author:Jessica Lynn
Post published:March 26, 2024
Post category:Systemic Scleroderma Systemic Sclerosis

Right now, no cure exists for people living with systemic sclerosis (also known as systemic scleroderma). There are treatment options available to manage systems. However, these also come with issues.…

Rare Community Profiles: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide

Post author:Jessica Lynn
Post published:March 26, 2024
Post category:Barth Syndrome

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.…

SADS Foundation Will Meet with FDA in June on New Treatments for LQTS and CPVT

Post author:Jessica Lynn
Post published:March 25, 2024
Post category:Long QT Syndrome

The Sudden Arrhythmia Death Syndromes (SADS) Foundation - a Patient Worthy partner - was founded in 1991 to provide support and care for people affected by heart arrhytmia conditions that…

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American College of Medical Genetics and Genomics 2024: Advances Promise a Better Future for Rare Disease Patients

Post author:James Moore
Post published:March 25, 2024
Post category:Rare Disease

The overwhelming majority of rare diseases and conditions have a genetic basis. This means that the disease is the result of a genetic abnormality such as a mutation. These abnormalities…

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Strength Beyond Measure: NFL Hopefuls Bench Press for Rare Disease Awareness

Post author:Jessica Lynn
Post published:March 25, 2024
Post category:Rare Disease

In the world of sports, the spotlight often shines on athletic prowess and competitive spirit. Yet Uplifting Athletes—a Patient Worthy partner—has transformed the narrative on the power of sport for…

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New Podcast Episode: Hemophilia and Axel’s Story, feat. Patient Advocate Kristina Robinson

Post author:Colby Rogers
Post published:March 22, 2024
Post category:Hemophilia A

Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kristina Robinson, a…

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An International Team of Scientists Discovered How Pneumococcal Bacteria is Destroyed After Vaccination

Post author:Rose Duesterwald
Post published:March 22, 2024
Post category:Pneumococcal pneumonia Rare Disease

For years, scientists have attempted to discover what destroys pneumococcal bacteria after a person has been vaccinated. In a recent study, vaccinologists in the U.S., China, and Switzerland, found the…

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FDA Approves Tyenne, an Actemra Biosimilar for Autoinflammatory Diseases

Post author:Jessica Lynn
Post published:March 22, 2024
Post category:Giant-cell arteritis Juvenile idiopathic arthritis Rheumatoid Arthritis

In early March 2024, Tyler Patchen reported that the U.S. Food and Drug Administration (FDA) approved both intravenous and subcutaneous formulations of Tyenne (tocilizumab-aazg) for use in a variety of…

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Rare Classroom: Narcolepsy

Post author:James Moore
Post published:March 22, 2024
Post category:Narcolepsy

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…

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The Rising Threat of Colorectal Cancer and the Importance of Early Screening

Post author:James Moore
Post published:March 21, 2024
Post category:Colorectal cancer

Rates of colorectal cancer are on the rise. In the US and other parts of the developed world, people seem to be developing this cancer earlier in life, for reasons…

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March is Multiple System Atrophy Awareness Month

Post author:Rose Duesterwald
Post published:March 21, 2024
Post category:Multiple system atrophy

Researchers worldwide spend years studying various aspects of a disease searching for a cure with help initially from public and private sectors. The primary source of the funding is usually…

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NORD State Report Card: Each State Graded on Rare Disease Care Access

Post author:Jessica Lynn
Post published:March 21, 2024
Post category:Rare Disease

Since 2015, the National Organization for Rare Disorders (NORD) has released an annual State Report Card that highlights state policies which could affect how people with rare diseases access care.…

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Rare Community Profiles: How Patient Advocate Kecia J. Survived and Thrived Through Her Rare Colorectal Cancer Battle

Post author:Jessica Lynn
Post published:March 20, 2024
Post category:Colorectal cancer Timely

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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FDA Approves Gene Therapy Lenmeldy for Metachromatic Leukodystrophy (MLD)

Post author:Jessica Lynn
Post published:March 20, 2024
Post category:Metachromatic Leukodystrophy

A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…

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Living with Cyclic Vomiting Syndrome: Josh’s Story

Post author:Jessica Lynn
Post published:March 20, 2024
Post category:Cyclic Vomiting Syndrome

When Josh Wright, 13, was younger, he always seemed to be somewhat of a sickly child. His mother Julie remembers Josh's first hospitalization at just one year old; her…

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Rare Community Profiles: In Pursuit of Progress: From HSP Diagnosis to Rare Disease Research

Post author:Jessica Lynn
Post published:March 20, 2024
Post category:Hereditary Spastic Paraparesis

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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FDA’s Refusal to Review Rare Disease Treatment Denies Patients’ Right to Try

Post author:Patient Worthy Contributor
Post published:March 19, 2024
Post category:Barth Syndrome

This opinion piece was provided by Kevin Woodward of Phoenix, Maryland. Kevin is a member of the Barth Syndrome Foundation board of directors. The role of the U.S. Food and…

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New Podcast Episode: The Road to Resilience and Self-Advocacy, feat. Patient Advocate Kecia J.

Post author:Colby Rogers
Post published:March 19, 2024
Post category:Colorectal cancer

Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. This week, Colby is sitting down with Kecia J, an…

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UK Approves Ztalmy (Ganaxolone) for CDKL5 Deficiency Disorder

Post author:Jessica Lynn
Post published:March 18, 2024
Post category:CDKL5

On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…

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March is National Kidney Month!

Post author:Jessica Lynn
Post published:March 18, 2024
Post category:Chronic Kidney 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…

Pitolisant for Prader-Willi Syndrome (PWS) Earns Orphan Drug Designation

Post author:Jessica Lynn
Post published:March 14, 2024
Post category:Prader-Willi Syndrome

Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…

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PGN-EDO51 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

Post author:Jessica Lynn
Post published:March 14, 2024
Post category:Duchenne Muscular Dystrophy

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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March 23: Lee University to Host the 2024 65 Roses 5K for Cystic Fibrosis (CF) Awareness

Post author:Jessica Lynn
Post published:March 14, 2024
Post category:Cystic Fibrosis

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: A Potential Treatment Turning Point for ALK-Positive NSCLC: Dr. Ken Culver Shares Insights from the Phase 3 ALINA Study on Alecensa

Positive Data Suggests NT-0796 Could Benefit People with Parkinson’s Disease

CABA-201 for Systemic Sclerosis Earns Orphan Drug Designation

Rare Community Profiles: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide

SADS Foundation Will Meet with FDA in June on New Treatments for LQTS and CPVT

American College of Medical Genetics and Genomics 2024: Advances Promise a Better Future for Rare Disease Patients

Strength Beyond Measure: NFL Hopefuls Bench Press for Rare Disease Awareness

New Podcast Episode: Hemophilia and Axel’s Story, feat. Patient Advocate Kristina Robinson

An International Team of Scientists Discovered How Pneumococcal Bacteria is Destroyed After Vaccination

FDA Approves Tyenne, an Actemra Biosimilar for Autoinflammatory Diseases

Rare Classroom: Narcolepsy

The Rising Threat of Colorectal Cancer and the Importance of Early Screening

March is Multiple System Atrophy Awareness Month

NORD State Report Card: Each State Graded on Rare Disease Care Access

Rare Community Profiles: How Patient Advocate Kecia J. Survived and Thrived Through Her Rare Colorectal Cancer Battle

FDA Approves Gene Therapy Lenmeldy for Metachromatic Leukodystrophy (MLD)

Living with Cyclic Vomiting Syndrome: Josh’s Story

Rare Community Profiles: In Pursuit of Progress: From HSP Diagnosis to Rare Disease Research

FDA’s Refusal to Review Rare Disease Treatment Denies Patients’ Right to Try

New Podcast Episode: The Road to Resilience and Self-Advocacy, feat. Patient Advocate Kecia J.

UK Approves Ztalmy (Ganaxolone) for CDKL5 Deficiency Disorder

March is National Kidney Month!

Pitolisant for Prader-Willi Syndrome (PWS) Earns Orphan Drug Designation

PGN-EDO51 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)

March 23: Lee University to Host the 2024 65 Roses 5K for Cystic Fibrosis (CF) Awareness

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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