Vision loss can be challenging. If you could have a one-time gene therapy that would improve your visual acuity, would you take it? It seems like a no-brainer. That's what…
While there is no cure for myasthenia gravis, this disorder can be managed with treatment. Current treatment options include a thymectomy, C5 protein inhibitors, and intravenous immune globulins (among others).…
Over the past few years, Akero Therapeutics has experienced high highs and low lows in its efforts to advance efruxifermin, an investigational drug for people living with metabolic dysfunction-associated steatohepatitis…
There are limited therapeutic interventions for those living with Niemann-Pick disease type C (NPC), a rare and progressive genetic disorder. Unfortunately, many people with NPC experience severe and life-threatening complications…
Dupixent has long been known as a therapy which reduces itch, slows disease progression, and reduces exacerbations in a number of allergic conditions such as asthma, eczema, and nasal polyps.…
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…
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.…
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…
Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which donor immune cells attack the recipient's tissues. The graft cells recognize…
In February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
In early March 2024, Clinical Trials Arena reports that global biopharmaceutical company Bristol Myers Squibb shared new data from the Phase 3 DAYBREAK study. Within the study, researchers explored how…
The 20th annual WORLDSymposium took place from February 4-9, 2024. During the course of the research conference, at least one presentation centered on data from the Phase 2 Mini-COMET long-term…
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
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…
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…
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…
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…
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.…
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…
Chronic spontaneous urticaria (hives) - an itch that can't be scratched. Well, from a literal perspective, you can scratch. But the chronic nature of chronic spontaneous urticaria means that your hives will…
In December 2023, pharmaceutical and biotechnology company Moderna announced that results from a Phase 2/3 clinical trial were published in The New England Journal of Medicine. The study explored an mRNA-based…
In December 2023, clinical-stage biopharmaceutical company Clene Nanomedicine ("Clene") shared that it had met with the U.S. Food and Drug Administration (FDA) regarding Accelerated Approval for CNM-Au8®, an experimental therapy…
In the United States, companies must obtain approval from the U.S. Food and Drug Administration to run clinical studies and transport or distribute therapies across state lines. To do so,…
In the past, Hyqvia [immune globulin infusion 10% [human] with recombinant human hyaluronidase] was approved to treat people with primary immunodeficiencies (PIs) ages two and older. Now, following data from…
Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet…
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