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…
An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
Right now, there are only an estimated 10 people in Ireland who have been diagnosed with a form of mucopolysaccharidosis type I (MPS I) called Hurler syndrome. This is the…
Trigeminal neuralgia is characterized by intense and debilitating chronic pain affecting one side of the face. Currently, there are a number of treatment options to reduce pain: tricyclic antidepressants, anticonvulsants,…
When she was an infant, Phoenix Radziwon was diagnosed with Down syndrome. However, her family has — over more recent years — learned that her Down syndrome diagnosis may go…
In March 2022, T. Kavinraj began experiencing a number of worrying health symptoms. He had a splitting headache and could not stop vomiting. In fact, his condition was so bad…
This year, the 13th annual Moebius Syndrome Awareness Day (MSAD) will take place on Tuesday, January 24. Moebius Syndrome Awareness Day, as the name suggests, is an event to celebrate…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
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…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
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