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…
In a late May 2023 news release from clinical-stage biotechnology company Acepodia, the company shared that the first patient has been dosed in a Phase 1 study. Within this…
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…
The AACR Annual Meeting 2023 took place from April 14-19. During the meeting, stakeholders from across the cancer community—from patients to clinicians—come together to discuss trends, research, and clinical practice.…
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics in conjunction with Chiesi Global…
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 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…
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…
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…
According to a story from Endpoints News, the drug developer Incyte has opted to put an end to its phase III clinical trial evaluating parsaclisib, an orally available PI3K inhibitor,…
In a news release from mid-April 2023, clinical-stage biotechnology company Abivax SA shared that 2-year results were available from a Phase 2b open-label maintenance study evaluating obefazimod for individuals…
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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