The advocacy group Just Treatment recently published a press release titled 'BREAKING: Cystic Fibrosis Patients Launch Global Challenge to Vertex Monopoly on CF Drugs.' Vertex Pharmaceuticals has taken the lead…
This week the family of actor Bruce Willis announced that he received a diagnosis of frontotemporal dementia (FTD), a rare disease that causes behavioral changes, language, speech, and memory…
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…
Previously known as Marburg hemorrhagic fever, Marburg virus disease (MVD) is a rare but serious viral hemorrhagic fever with sporadic outbreaks occurring in South Africa, Ghana, Zimbabwe, Uganda, Kenya,…
In the United States, Fast Track designation is granted to drugs designed to treat rare and/or serious conditions and that fill an unmet medical need. The process aims to…
February 15th is recognized annually as International Angelman Day, a day dedicated around the world to spreading awareness among the general public and in the medical field about Angelman syndrome,…
Four-year-old Rayhan Majid of North Lanarkshire, UK was a healthy little boy who enjoyed swimming, football, and Taekwondo. He began to have severe headaches in October 2017. His mum,…
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
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…
Known as “Slydah,” Chicago White Sox relief pitcher (RP) Liam Hendriks has done amazing things on the field. In September/October 2021, Hendriks was named the American League Reliever of…
ABC News recently carried an article describing an anticipated surge in the latter part of 2020 of acute flaccid myelitis. Thankfully, it did not occur. The disease is caused by…
In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia…
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…
Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
If you’ve ever wanted to run around Philadelphia in your underwear for a good cause, I have an event to tell you about! As first reported by the Philly…
In the reality television world, Megan Marx is best known for her time on Bachelor Australia, Bachelor in Paradise, and The Challenge Australia. But Marx is about to be…
Little 2 1/2-year-old Raiden Pham cannot walk or talk, but he can smile at his parents and radiate love. The baby’s parents are on a 24/7 schedule caring for…
Jenny Decker’s disease, Charcot-Marie-Tooth (CMT), is named after the three doctors who first discovered the disease in 1886. CMT is a progressive neurological disease that affects approximately 2.6…
Jason Tolson age 30 of Yorkshire, UK had not been diagnosed when he first began to have symptoms in May 2022. Jason began to experience muscle and joint pain…
It has historically been difficult to encourage the development of therapies for patients with rare diseases. To overcome this, the Orphan Drug Act was passed in 1983. This act…
Genethon is a non-profit organization committed to the design and development of gene therapies for rare diseases. This unique company is a pioneer in identifying genes relating to genetic…
Have you ever heard of Orphan Drug designation? This designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics intended to diagnose, prevent, or…
Generalized arterial calcification of infancy (GACI) and autosomal recessive hypophosphatemia rickets type 2 (ARHR2) Awareness Day is recognized each year on Feb 1. This is a time for spreading awareness…
Prematurity and formula-feeding heighten the risk of necrotizing enterocolitis (NEC), a serious gastrointestinal issue. However, NEC is still poorly understood; in many cases, the exact cause is still unknown.…
Doddie Aid, which begins January 1st and continues for the following six weeks, aims to encourage people to get active while raising funds to support research into motor neuron disease…
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