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.
In June 2022, Andrew Mace received CPR from a neighbor, 21-year-old Alex Duncan, a med student who luckily remembered her instructions while at Cambridge University, UK. Andrew, age 44,…
The failure rate of drug development for Alzheimer’s is 99 percent according to a recent study. Since 1984 scientists have concentrated on developing treatments to prevent the formation of…
Emergency Room (ER) doctors are gradually being replaced by physician assistants and nurse practitioners. The Neiman Institute reported that between 2005 and 2020 the number of ER visits with midlevel…
A town hall meeting was held on February 7th of this year to discuss designs for clinical trial gene therapies. According to a report in Pharmaceutical-Technology, the FDA experts were…
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…
509 words 8% matched vs 786 words 5% matched A new study recently appeared in the publication The Conversation written by authors Laura Whitworth, Group Laboratory Manager, and Prof. Lalita…
Researchers are closing in on cancer from every angle. Recently a team at the Colorado University (CU) Cancer Center has been focusing on p53, a common gene mutation found…
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.…
The Schleswig-Holstein University Hospital and the Max Planck Institute in Germany have investigated a hereditary condition that is extremely rare called brachyphalangy, polydactyly, and tibial aplasia/hypoplasia (BPTA) syndrome. Symptoms…
Two Southern California University studies showed a reduction of neurodegenerative activity in amyotrophic lateral sclerosis (ALS) research models with each using a different treatment route. As reported in Inside Precision…
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…
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