Three organizations focused on amytrophic lateral sclerosis (ALS) as well as other rare neurodegenerative diseases, have just celebrated the House of Representatives' passage of the ACT for ALS (H.R.3537/S.1813). The…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Researchers from both Kumamoto University in Japan and the Imperial College in London have recently completed a research study in adult T-cell leukemia/lymphoma (ATL), a rare type of leukemia. They…
According to Healio, the U.S. Food and Drug Administration (FDA) recently granted Fast Track designation to LioCyx-M004. This therapy, developed by T-cell immunotherapy company Lion TCR, is designed to…
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
In the European Union (EU), Orphan Drug designation is given to drugs or biologics intending to improve the treatment, diagnosis, or prevention of rare or serious illnesses. To be…
Continued From Part One Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering.…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this status, “rare” conditions…
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…
Clinical trial results can help support drug developers' goals. For example, positive trial results may support Marketing Authorization Applications (MAAs) or other areas needed to get therapies into the…
January has been designated as a time to bring awareness to alternating hemiplegia of childhood! Alternating hemiplegia of childhood is extremely rare, with about 1 in 1,000,000 reported cases; therefore…
Kyleigh Kista, a 3 year old from Benonin South Africa needs heart surgery within the next 3 months to save her life. Kyleigh was diagnosed with tetralogy of Fallot with…
The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, "rare" conditions are those affecting…
According to a recent article, the preliminary results of a study suggest that certain oral and infusion therapies may be beneficial for those suffering from fatigue due to myalgic…
Life is filled with endless ups and downs. The first 5 of my ongoing 20 years living with dystonia were pretty much nothing but down times. I experienced the most horrible pain…
Pursuing an education can be difficult enough without any other extraneous factors. But what if you were also battling frequent infections, gastrointestinal problems, and recurrent hospitalizations? For Evan Brandon,…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
Prometheus Biosciences Inc. has just announced positive results from their Phase 1 investigation for a new treatment for systemic sclerosis-associated interstitial lung disease (SSC-ILD). This trial investigated PRA023, an anti-TL1A…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
Catherine Nettles Cutter, a professor at Penn State University, vividly recalls two years of her life spent trying to recover from a bodysurfing accident that resulted in the mysterious loss…
Over the last 10+ years, there have been extreme advances in the treatment of metastatic melanoma. In particular, researchers have sought to develop therapies which halted cancer progression and reduced…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
According to a recent article, patients with phenylketonuria (PKU) are unable to eat proteins because of their condition. However, a new probiotic might be the solution. Phenylketonuria (PKU) PKU is…
Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…
A retrospective medical records study for interstitial lung disease (ILD) has just been published in Arthritis Care & Research. The results found that for women diagnosed with ILD as well as an…
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