At the beginning of our interview, Lindsey Curley explains to me that there is a difference between listening and understanding. Listening is important, of course; she wants people to listen…
According to a recent article, the European Society of Cardiology has recommended that athletes undergo gene testing to prevent sudden cardiac death from exercise. Gene Testing Lethal variants can be…
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. Compassion Corner is a…
According to a recent article in The British publication The Economic Times, a group of researchers at McMaster University have a win-win proposal for myotonic dystrophy (MD) patients. Eleven people were…
Getting a rare disease diagnosis can be difficult and take a long time. This can be a problem, as time is something that those with progressive illnesses don't have much…
According to a recent article, a new study where SIOD patients had a dual immune/solid organ transplant yielded positive results with patients accepting their new kidneys without rejection. Schimke Immune-Osseous…
According to a recent press release from Biospace, the first cohort of the ReSPECT-LM trial has officially completed enrollment. This trial focuses on Rhenium-186 NanoLiposome (186RNL) as a treatment for…
Medscape has recently reported on a new supplemental approval from the FDA for setmelanotide, which is a treatment for obesity associated with Bardet-Biedl syndrome (BBS). Now, BBS patients who are…
The month of June is recognized as Intrahepatic Cholestasis of Pregnancy (ICP) Awareness Month, a time to spread awareness about the rare condition among the general public and the medical…
Have you ever awoken in bed and thought that what happened the day before must have been a nightmare? It CANNOT possibly be true that I have a rare disease…
The future of healthcare is going to be changed and impacted by the use of artificial intelligence (AI), hopefully in beneficial ways. According to an article from McMaster University, researchers…
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…
This year, the Congress of Clinical Rheumatology East took place from May 12-15, 2022. During the conference, clinical rheumatologists gathered to discuss trends in rheumatology, practice guidelines, and cutting-edge therapeutics.…
Kelly Heger, founder of the AADC Family Network, shares her story of becoming a nurse to take care of her daughter with AADC deficiency. When my daughter, Jillian, was born…
Cogent Biosciences has just released promising initial data from their Phase 2 clinical trial called APEX. This trial is investigating bezuclastinib, a selective KIT D816V inhibitor, as a treatment for…
Inherited retinal diseases (IRDs) are a group of genetic ocular disorders characterized by vision loss or blindness. A few examples are retinitis pigments (RP) and Usher syndrome. There has been…
Popular Canadian singer Justin Bieber stunned the world when he announced he was diagnosed with Ramsay Hunt syndrome following his facial paralysis. Ramsay Hunt Syndrome Ramsay Hunt syndrome (herpes zoster…
Thanks to Amylyx Pharmaceuticals, Canada becomes the first country worldwide to have access to an ALS drug branded Albrioza (originally AMX0035). According to an article in Yahoo News, the drug…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a…
Meghan Rourke saw the need for more open conversations about chronic illnesses, sparking her to write The Invisible Kingdom: Reimaging Chronic Illness. In an excerpt from her novel, she shares…
From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…
In clinical trials evaluating Zolgensma for children with spinal muscular atrophy (SMA), the maximum weight of participants was 18.7 pounds. However, parents with children weighing over that weight might be…
In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…
At just two days old, Leita’s son Glyn started having infantile spasms. For the next sixteen years, Glyn’s seizures worsened. Leita also had two other children – twins named Asha…
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…
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