Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
In 2022, the U.S. Food and Drug Administration (FDA) approved DAXXIFY® (DaxibotulinumtoxinA-lanm) for the temporary treatment of fine lines and wrinkles. Many people touted DAXXIFY as a "Botox alternative."…
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
As recently reported by Forbes, the U.S. Food and Drug Administration (FDA) approved Leqembi (lecanemab) for Alzheimer’s disease earlier this week. Leqembi is a humanized monoclonal antibody that targets…
In late June 2023, global biopharmaceutical company UCB announced that the U.S. Food and Drug Administration (FDA) approved RYSTIGGO (rozanolixizumab-noli) for adults living with AchR or MuSK antibody positive…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
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…
Rinvoq (upadacitinib) is a prescription JAK inhibitor that works by blocking pro-inflammatory signaling within cells. In the past, Rinvoq was approved for indications like rheumatoid arthritis, active psoriatic arthritis and…
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…
In the past, budesonide has been used in a number of therapeutic indications, such as to reduce proteinuria in IgA nephropathy or digestive tract inflammation in those with Crohn's disease.…
According to reporting for Cure Today, the FDA recently granted Accelerated Approval to a combination treatment of Padcev (enfortumab vedotin-ejfv) and Keytruda (pembrolizumab) for advanced or metastatic urothelial carcinoma.…
An estimated 6,000-10,000 people within the United States are living with Rett syndrome. Prior to this month, these individuals have not had access to targeted treatment options. But this…
While there are existing therapies for individuals living with homozygous familial hypercholesterolemia (HoFH), a rare form of high LDL cholesterol, these therapies often fail to control cholesterol levels for…
Prior to February 2023, there were no FDA-approved treatment options for non-central nervous system manifestations of alpha-mannosidosis. But in mid-February 2023, Chiesi Global Rare Diseases shared via news release…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
Until recently, the only therapeutic options for people with Friedreich ataxia were symptomatic and focused on symptom management: mobility aids, surgery, psychotherapy, speech therapy, heart medications, hearing and vision…
In the past, there have been limited therapeutic options for pediatric patients living with hereditary angioedema (HAE). Children aged 2 to <6 years old had no approved prophylaxis therapy. For…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
Currently, C5 inhibitors such as eculizumab and ravulizumab are considered the standard-of-care for people living with paroxysmal nocturnal hemoglobinuria (PNH). Unfortunately, not all patients respond well to these therapies.…
1On July 27, 2022, global biopharmaceutical company GSK plc shared some exciting news via news release. The company’s therapy Benlysta (belimumab), designed for pediatric patients with lupus nephritis, is now…
Will teclistamab soon be available for patients with multiple myeloma within the European Union? According to OncLive, approval seems to be on the horizon. Following results from the Phase ½…
In the past, filgotinib (Jyseleca) has largely been used to treat patients with rheumatoid arthritis (RA). However, researchers hypothesized that the treatment could be beneficial in other indications. Following…
There are three subsets of Von Willebrand disease (vWD), a genetic bleeding disorder. Type 3 is considered to be the most severe form of vWD, culminating in intense or severe…
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