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.
A study that took place between 2003 to 2013 reported 8,243 preventable deaths in Wales and England. All indications pointed to the poor quality of care that women received in…
Jimmy Buffett survived a 1994 plane crash that was the inspiration for his song “Live Like It’s Your Last Day." According to an obituary on his website, Jimmy, 76,…
As reported by MSN, researchers have discovered that the blood vessels feeding aggressive brain tumors can be useful. The blood vessels have receptors that contain nanoparticles that can be…
Novartis AG, with corporate headquarters based in Basel, Switzerland, recently announced positive results from its Phase III trials REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) ). The news was delivered via Drugs.com.…
Social media has become a vehicle for customer complaints and the companies are listening. Estimates are that almost two-thirds of dissatisfied customers have received a response to their online…
A team of surgeons at the Langone Institute performed its fifth animal-to-human transplant using organs from genetically modified pigs. The Revivicor company modified the organ in order to prevent rejection…
When a family member is unable to care for themselves, proper training is essential for all parties involved. A case in point was the crisis Patti LeFleur was unable…
65 million people in the U.S. who are on Medicare are prescribed medications according to a mid-year KFF healthcare survey. 60% of these individuals are prescribed a minimum of…
Accutar Biotechnology, Cranbury, New Jersey recently announced via BioSpace that dosing has begun with the first patient enrolled in the Phase 1 trial of AC0676 a chimeric degrader molecule…
According to a press release from Verrica Pharmaceuticals and its partner Lytix Pharmaceutical via Healio, results of Part 1 of the phase 2 study of their investigational therapy candidate VP…
A 15-year search for a diagnosis by Lilly Grossman and her parents ended in 2013. Lilly became the first person in the world to be diagnosed with ADCY5-related dyskinesia.…
A recent press release from Verrica Pharmaceuticals and its partner Lytix Biopharma reported in Healio announces the results of the first portion of a study evaluating VP-315 to treat basal…
The life of the four-year-old was saved. The operation conducted at Medcare Orthopedics was a success. It was the first of its kind to be performed in the UAE.…
Enterome presented its findings to the 17th International Conference on Malignant Lymphoma (ICML) in June of this year. As reported in the GlobeNewswire, the company demonstrated that E02463 together…
The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…
It was the second time 26-year-old Bruce Campbell had to deal with paralysis. At the age of six, he was paralyzed by Guillian-Barre syndrome which caused an attack on…
Sounds like ‘food for thought’. The term ‘sandwich generation’ describes family members who are usually between the ages of 45 and 55 and are responsible for raising their children…
The number of novel drugs approved by the FDA has doubled in the ten-year period from 2012 through 2020. Investment in investigational drugs for rare diseases appears to be continuing…
How Jenny’s Voyage Was Salvaged BigIslandNow recently carried the story of how Jenny’s dream was almost shattered when she was sold a $10,000 used engine rather than an $8,000 new…
Kona nurse Jenny Decker, 38, left Honokōhau Small Boat Harbor, Kailua-in Hawaii on June 28, 2023. A recent article in BigIslandNow announces that Jenny is ready to circumnavigate…
Each summer advocates for rare diseases take advantage of longer daylight hours and use the opportunity to showcase the hopes, needs, and wants of people with rare diseases such as,…
The success of the CARES 310 Trial Phase III (NCT03764293 ) was reported this week in Biospace by Elevar Therapeutics. The combination of two drugs, rivocera, and camrelizumab proved to…
According to an article in MedicalXpress News Today, current research indicates that rare diseases in the United States affect 25-30 million people. The numbers keep rising and rival diseases…
The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
Professor Timothy Yu developed the custom drug milasen named in honor of Mila, an 8-year-old girl with Batten disease. The drug is the first drug specifically designed for one…
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