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.
Dozens of Ryan Wilson Palmer’s family and friends recently celebrated an early birthday after he received a devastating diagnosis of Creutzfeldt-Jakob disease. A few months ago 49-year-old Ryan Palmer of…
Continue ReadingRyan’s Life Changed from being a Fire Fighter to Being Diagnosed with Creutzfeldt-Jakob Disease
Antibody-drug conjugates (ADC) are one of the fastest-growing anticancer drugs. BioNTech’s licensed ADC is now in Phase III. According to a report in BioSpace, BioNTech is attempting to compete with…
Continue ReadingBioNTech and DualityBio Moving ADC into Phase III for HER2 Low Breast Cancer
Beam Therapeutics (Beam) has developed a technique called base editing. The treatment is called BEAM 201. The first patient has been dosed according to a recent statement by the company…
Continue ReadingBeam Therapeutics Announces a First in Human Testing of Gene Editing Medicine for Cancer
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…
Continue ReadingPreventable Deaths: Physicians are Dismissing Women’s Pain
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,…
Continue ReadingMerkel Cell Carcinoma Has Taken the Life of Jimmy Buffett After Fending Off Cancer for Four Years
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…
Continue ReadingExperimental Treatment Shows Promise in Diffuse Midline Glioma
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.…
Continue ReadingBTK Inhibitor Brings Rapid Improvement in Chronic Spontaneous Urticaria
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…
Continue ReadingPatients and Doctors Are Pressuring Health Insurance Companies Online in Order to Receive Proper Treatment
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…
Continue ReadingPig Kidney Survives in a Human for Six Weeks and Beyond in New Milestone
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…
Continue ReadingNew Proposals Aim to Support Caregivers Through Medicare
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…
Continue ReadingMedicare Begins First Wave of Price Negotiations with Pharmaceutical Manufacturers
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…
Continue ReadingClinical Trial of Immunotherapy for Basal Cell Carcinoma Shows Promise
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…
Continue ReadingNon-surgical Immunotherapy Treatment Shows Promise in Basal Cell Carcinoma
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.…
Continue ReadingDubai Doctors Saved a Child with Moyamoya Disease with a Special Technique
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…
Continue ReadingEnterome Announces Positive Preliminary Trial Data for Non-Hodgkin Lymphoma
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…
Continue ReadingA Preview of Potentially Historic FDA Approvals for 2023
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…
Continue ReadingParalyzed with Pompe Disease, He Took Control, Gained Strength, and Increased Muscle
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…
Continue ReadingHybrid Work and the Sandwich Generation of Caregivers
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…
Continue ReadingAn Increase in the Development of Treatments for Rare Diseases
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…
Continue ReadingKona Nurse with Charcot-Marie-Tooth Disease Will Circumnavigate the Globe Solo: Part 2
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…
Continue ReadingKona Nurse with Charcot-Marie-Tooth Disease Will Circumnavigate the Globe Solo: Part 1
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…
Continue ReadingNew Drug Combo Improves Survival in Inoperable Liver Cancer Patients
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