Rare Disease
Gut Check: How Fecal Microbiome Metabolites Predict ICU Survival
A groundbreaking study has revealed that the balance of metabolites produced by gut microbes (measured from a simple stool sample) can predict the short-term risk
New Duchenne Drug Offers Hope to Young Patient Losing Ability to Walk
- July 18, 2025
Takeda’s Oveporexton: A Promising Step
FDA Approves Ekterly: A New Oral Option for Hereditary Angioedema Attacks
European Commission Approves Groundbreaking Therapy for Duchenne Muscular Dystrophy
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Duchenne Muscular Dystrophy
Brogidirsen and the Promise of Exon 44 Skipping: New Horizons in RNA Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating, inherited muscle-wasting disorder caused by mutations in the DMD gene, leading to an absence of functional dystrophin protein.
Rare Disease
Food as Medicine: Harnessing Nutrition to Combat Chronic Disease
The concept of “food as medicine” has gained significant traction in recent years, and for good reason: mounting evidence shows that nutrition is a powerful
Multiple Myeloma
A Potential Turning Point: CAR T-Cell Therapy Shows Long-Term Survival in Advanced Multiple Myeloma
At the recent American Society of Clinical Oncology (ASCO) annual meeting, researchers unveiled remarkable long-term results for ciltacabtagene autoleucel (cilta-cel; Carvykti), a CAR T-cell therapy,
Diabetes Type 2
Breakthrough Cell Therapy Restores Insulin Production in Majority of Diabetics
A groundbreaking advance in diabetes care has emerged from recent clinical research: a new cell therapy has successfully restored natural insulin production in 83% of
Duchenne Muscular Dystrophy
Real-World Outcomes of Ataluren in Duchenne Muscular Dystrophy: Evidence from the STRIDE Registry
Duchenne Muscular Dystrophy (DMD) is a severe, progressive neuromuscular disease that primarily affects boys, leading to muscle weakness, loss of ambulation, and premature death. Among
Rare Disease
June 2025 FDA Oncology Approvals: Expanding Options and Personalization for Cancer Patients
June 2025 marked a month of significant progress in cancer care, as the FDA approved several new therapies and treatment combinations across a range of
Duchenne Muscular Dystrophy
Achieving Global Excellence in Duchenne Muscular Dystrophy Care: The Accredited Duchenne Centers Program
The Accredited Duchenne Centers (ADC) Program, spearheaded by the World Duchenne Organization (WDO), is a pioneering global initiative designed to standardize and elevate care for
Cancer
Analyzing the benefits of Regular exercise for Cancer Patients
For decades exercise has been recommended to improve the quality of life. More recently and perhaps more importantly, exercise has been highly recommended for cancer
Hereditary angioedema
KalVista Secures FDA Approval for First-Ever Oral On-Demand HAE Treatment
KalVista Pharmaceuticals has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of its new oral therapy for hereditary angioedema (HAE),
Macular degeneration
First Patient Dosed in Clinical Trial for Promising Dry AMD Therapy PST-611
A new milestone has been reached in the search for effective treatments for dry age-related macular degeneration (AMD), as the first patient has now been
Biliary tract cancer
FDA Grants Orphan Drug Status to FF-10832 for Biliary Tract Cancer
Fujifilm Corporation’s investigational therapy FF-10832 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of biliary tract cancer,
Chronic obstructive pulmonary disease (COPD)
The Danger of Misdiagnosing COPD
Editor’s Note: This article was originally published on 3/5/25, and is being republished due to ongoing efforts by Patient Worthy to raise awareness of COPD.
Neuroendocrine Tumor
From Pharma Rep to Patient Advocate: My Journey with Neuroendocrine Cancer
My name is Kelly Baughman, and I grew up in a small town in Louisiana—tight-knit, faith-driven, and yes, I went to church and summer camp
Measles
US Measles Cases Surge Amid Vaccine Hesitancy and Political Debate
The United States is experiencing its worst measles outbreak in decades, with cases rising sharply across multiple states. Health officials attribute this surge primarily to
Hidradenitis suppurativa
Children with Hidradenitis Suppurativa Face High Rates of Acne, Obesity, and Anxiety
A recent study has brought attention to the complex challenges faced by children with hidradenitis suppurativa (HS), a chronic and often painful skin condition. According
Lung Cancer
Corticosteroids May Undermine Immunotherapy Success in Lung Cancer Patients
A new study has found that corticosteroids, commonly used to manage symptoms in lung cancer patients, may reduce the effectiveness of immunotherapy treatments. The research,
Rare Disease
Eosinophilic Gastrointestinal Diseases Linked to Higher Rates Among Patients with Allergies
Recent research highlights a significant connection between eosinophilic gastrointestinal diseases (EGIDs) and atopic conditions such as asthma, eczema, and allergic rhinitis. EGIDs, which include disorders
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