Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
Their son, Caeleb, was only eleven months old when he developed a severe bleeding episode. The family had just moved from Houston to New Mexico and they had not yet…
Kimberly Greenberg’s baby, Parker, was born a few weeks early but came into this world on January 2, 2016, as a healthy, normal baby. However, just before Parker was to…
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,…
Whole genome sequencing is providing high-level information about new rare diseases putting pressure on drug developers to pick up the pace. Of approximately 7,000 rare diseases, most do not have…
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…
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…
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…
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…
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 Rylae-Ann was one year old, her parents had to hold her in an upright position at night in order for her to breathe. Rylae-Ann had a rare genetic disease,…
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…
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…
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…
Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg…
Singer Celine Dion recently acknowledged that she has an extremely rare neurological disorder called stiff person syndrome (SPS). According to a recent article in The Metro, the disease occurs…
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…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
Mazidul Islam told the Daily Star that he had to watch his 14-year-old son Farid die and could do nothing to save him. Among other things, he regrets the fact…
An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The antidepressant, imipramine, was approved to…
Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a recent article published in BioSpace,…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare disease called Charcot-Marie-Tooth disease. Doctors…
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