Diagnosing amyloidosis (abnormal protein in tissue) is a complex process. Over eighty percent of amyloidosis patients are undiagnosed. The disease takes hold after the blood plasma cells in the bone…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
The Pulmonary Hypertension Association recently announced it is offering junior investigators who are interested in pulmonary arterial hypertension (PAH) a two-year grant. Pulmonary Arterial Hypertension (PAH) Pulmonary arterial hypertension (PAH)…
PTC Therapeutics has just announced that their therapy Waylivra (volanesorsen), a treatment for familial chylomicronemia syndrome (FCS), has been given Category 1 classification from the Drug Market Regulation Chamber in…
Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…
It’s January 2022 – have you checked on your thyroid health lately? If not, consider learning more about thyroid disease and what your thyroid does for the body. Why…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
The largest study of benign adrenal tumors has just been completed. Results have been published in the Annals of Internal Medicine. Specifically, this study examined the effect of mild autonomous cortisol…
Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon. This treatment is a combination therapy which…
Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
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…
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