Currently, there are two FDA-approved treatments for people living with idiopathic pulmonary fibrosis (IPF): Ofev (nintedanib) and Esbriet (pirfenidone). While these therapies are effective in improving lung function, they…
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…
The 91st European Atherosclerosis Society (EAS) Congress took place from May 21-24, 2023. During the Congress, many stakeholders discussed the latest developments in basic, translational, and clinical research into vascular…
Ventilatory assistance, NG tubes, tracheostomy: all of these may be used to care for infants born with bronchopulmonary dysplasia (BPD), a chronic lung disease that most commonly affects preterm…
There are no cures or treatments available for people with hereditary spastic paraplegia (HSP) type 56, or SPG56. While the current standards-of-care focus on symptom relief and mobility assistance, patients…
First described in medical literature in 1949, Alexander disease is an extremely rare neurological disorder and form of leukodystrophy. Despite nearly 74 years having passed since its discovery, there is…
Within the Phase 3 APHENITY study, researchers worked to determine the efficacy of sepiapterin in both adults and children with phenylketonuria (PKU). Formerly known as PTC923, sepiapterin was developed…
In 2014, the National Heart, Lung, and Blood Institute released updated clinical guidelines in reference to the care of children with sickle cell anemia. The updated guidelines recommended the use…
Our country has an issue when it comes to screening for diabetic retinopathy. In addition to clinics lacking the necessary imaging programs, the compliance rate for diabetic retinopathy screening…
Have you ever thought about your genes and what they do for your body? For example, the KRAS gene—do you know what it does? Normally, KRAS encodes for the production…
3-year-old Ava Bolton has always been bright, vivacious, and gregarious. So her parents Natalie and Scott were understandably worried when, near her third birthday, Ava's affect seemed to change. According…
In a late April 2023 news release, biotechnology company UNITY Biotechnology, Inc. ("UNITY") shared that the company had observed positive long-term follow-up data from the Phase 2 BEHOLD trial. Within…
According to an article published in Cold Agglutinin News, Dianthus Therapeutics (“Dianthus”) is planning on holding a proof-of-concept study in 2024. Within the study, the company would evaluate the safety…
People with severe cases of Eastern equine encephalitis (EEE) or Venezuelan equine encephalitis (VEE) can develop significant brain inflammation, causing damage. An estimated 33% of people with severe EEE infection,…
This weekend during the Leiden Marathon, more than 30 parents, children, doctors, researchers and members of the public will run to raise money for the Well Bee Study. This important…
In the past, doctors and researchers surmised that having chronic kidney disease (CKD) could increase the risk of having a stroke. Yet they didn’t know exactly why it happened or…
Usher syndrome is a rare genetic disorder that is characterized by deafness and often paired with retinitis pigmentosa (RP), an inherited disease which causes retinal degeneration and vision loss. There…
During the REPREVIO clinical study, researchers sought to understand whether using vedolizumab (Entyvio) after bowel resection could prevent the risk of recurrence in individuals with advanced Crohn’s disease. Participants were…
There are still many unknowns when it comes to our genetics. How do gene variants of unknown significance (VUS) affect us? In what ways can we identify if variants…
KMT2A rearrangements—a DNA fault—lead to treatment-resistant acute lymphoblastic leukemia (ALL) in infants. While around 50% of children with KMT2A rearrangements respond well to intensive chemotherapy, the remaining 50% do…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
Each year, an estimated 20,100 people across America die from non-Hodgkin’s lymphoma (NHL). While treatment is available—namely chemotherapy and immunotherapy—researchers are always exploring new treatment options to help those affected…
Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
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