The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
When biopharmaceutical company Invex Therapeutics (“Invex”) first decided to launch a clinical trial to evaluate Presendin (sustained-release exenatide) for idiopathic intracranial hypertension (IIH), the company felt excited. Presendin is a…
An estimated 25% of people in the United States have a condition called nonalcoholic fatty liver disease (NAFLD), a condition characterized by excess fat in the liver. Estimates vary but…
According to a story from Healio, in a phase 3 clinical trial, treatment with the drug resmetirom was able to achieve the desired endpoints at 52 weeks in people living…
Since 2021, biotechnology company Foghorn Therapeutics ("Foghorn") has been developing its therapeutic candidate FHD-286 for a number of malignancies. In one Phase 1 study, researchers are testing the pharmacokinetics,…
Roivant Sciences recently shared updates on the Phase 2b TUSCANY-2 study. Within the study, the team was evaluating RVT-3101 for moderate-to-severe ulcerative colitis. RVT-3101 is a fully human monoclonal…
Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
Do mast cells—a type of white blood cell that plays a part in immune and allergic responses—contribute to the development of eosinophilic esophagitis (EoE)? In the past, eosinophils (another…
Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent…
Your HTT gene encodes for the production of huntingtin, a protein that helps your neurons (nerve cells in your brain) function correctly. When mutations happen in this gene, the protein becomes…
Clinical trials are incredibly meaningful in advancing research, understanding, and potential treatments for many conditions. Unfortunately, these trials are not always successful, meaning that the evaluated drugs cannot be considered…
uniQure, a leading gene therapy company, recently shared an update on a Phase 1/2 clinical study evaluating AMT-130 for Huntington’s disease. The safety and proof-of-concept study enrolled 26 participants with…
The European Congress of Rheumatology (EULAR) took place from May 31 - June 3, 2023. During the Congress, experts in rheumatology came together to discuss research, new guidelines, and…
There are current approved therapies for people living with wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). For example, doctors may prescribe VABYSMO, Lucentis, Beovu, Avastin,…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Right now, there are insufficient treatment options for familial chylomicronemia syndrome (FCS). Unfortunately, this means that individuals living with this rare genetic disease are left without much assistance. The…
Have you ever had an itch that you just can't seem to scratch? Intensely itchy skin, also known as pruritus, is a hallmark characteristic of many conditions that affect the…
Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. The…
Sometimes it can be difficult to talk about our health. For people living with genital cancers, there are sometimes additional burdens: a fear of discussing a "taboo" topic, concerns…
About 150,000 people with glaucoma across the United States wear contact lenses—and they may soon have access to an even more effective wearable treatment option. According to Medical Device Network,…
In early 2022, the U.S. Food and Drug Administration (FDA) approved Carvykti (ciltacabtagene autoleucel) for the treatment of patients with multiple myeloma. Carvykti is a BCMA-directed T cell immunotherapy.…
When it comes to finding novel ways to treat or cure cardiovascular diseases, Cereno Scientific is committed to making change. The clinical-stage biotechnology company works in the realm of both…
In August 2022, I reported on a story that touched my heart: Lucy Landman's PGAP3 diagnosis. With less than 50 diagnosed cases of PGAP3 worldwide, parents Zach and Geri jumped…
The American Society of Clinical Oncology (ASCO) held its Annual Meeting from June 2-6, 2023. During the meeting, a variety of stakeholders discussed clinical practices, guidelines, trends, and research related…
Children born with propionic acidemia require life-long treatment and care; without it, this disorder can lead to coma or even death. While there are supportive treatment options, no current…
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