Prior studies have suggested that metformin, often used as a first-line treatment for people with type 2 diabetes (T2D0, could confer benefits for Rett syndrome. In particular, prior studies found…
Lauren Bruccoleri and her husband Matthew weren’t initially planning on having their son Grayson. But when Lauren found out that she was pregnant, she was overjoyed. However, Lauren and Matthew…
When Kandise MacLeod was twelve years old, she began noticing various growths and tumors popping up on her bones. These sometimes caused pain or discomfort; in one case, Kandise even…
Seizures associated with conditions such as Dravet syndrome and Lennox-Gastaut syndrome (LGS) can be difficult to treat; these seizures may be treatment-averse and may not respond well to current anti-epileptic…
Have you heard of the pulmonary microbiota, which may also be referred to as the lung microbiome? This refers to the complex collection of microorganisms (bacteria, viruses, bacteriophages, fungi) that…
Stat News recently published comments by Bruce Bloom, collaboration officer at Healx, Cambridge, UK heralding the major advances in the treatment and the study of rare disease during 2022.…
Before you read on, make sure to check out Part 1 of Kristin and Kayden's story. In Part 1, Kristin discusses the diagnostic journey and how Kayden was diagnosed with a dermal…
Patients want to be an integral part of drug development. Their voices have been heard on many topics ranging from the importance of Newborn Screening, choosing meaningful endpoints and meaningful…
A rare disease diagnosis can conjure up a multitude of emotions, from relief at finally learning what is going on to fear or isolation when trying to figure out what…
Before you read on, don't forget to check out Part 1 of Jennifer's story. In Part 1, Jennifer shared her diagnostic journey and the four year stretch it took her to receive…
Initially, belimumab (sold under the brand name BENLYSTA) was approved for the treatment of patients for lupus and lupus nephritis. It was the first FDA-approved biologic treatment within this indication.…
Gavin Chandler loves toy cars, watching Big City Greens, fans, the “Baby Shark” song, and anything that lights up or makes noise—including, his mother Jessica shares, the vacuum cleaner. He…
In the United States, Orphan Drug designation is granted by the FDA to drugs focused on the treatment, prevention, or diagnosis of rare conditions; these are those affecting fewer than…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
My name is Becky Tilley. I live in the UK, am married, and my wonderful husband Carl and I have three children: Isabella (age four), Joshua (age two), and Avary…
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…
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…
The 2023 Special Olympics BC Winter Games took place from February 2-4, 2023. Sporting events at Winter Games include 5-pin bowling, curling, figure skating, alpine or cross-country skiing, and more!…
Dr. Valeria Ricotti of the Institute for Child Health- Great Ormond Street UK, spoke with BBC News on the subject of AI analyzing body movement. Dr. Ricotti and other researchers…
On January 26, 2023, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
This is Part 2 of a two part story. Check out Part 1 here. The loss of his child through Edwards Syndrome helped Dr. Harsha Rajasimha understand the suffering and…
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…
PART I The baby died almost immediately after birth. Edwards Syndrome is a rare congenital disease. Only about ten percent of newborn babies survive past their first year. Babies…
When Arica Svoboda thinks of her two children, Hayes and Hendrix, she can’t help but fill up with love. She tells Patient Worthy: “My boys are special. Their smiles and…
Treatment for Alagille syndrome, a rare genetic disorder that affects multiple organ systems, are typically symptomatic. Many children born with this disorder eventually require a liver transplant; without this, Alagille…
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