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.
The FDA has approved Rystiggo, a developed by UCB in Brussels, to treat two types of myasthenia gravis. The approval was based on the results of the Phase III…
The incidence of gastroschisis has doubled over the past 20 years. Read “Jeanie’s Gastroschisis Baby” and learn from a brave, young first-time mom about discovery and how Gastroschisis can be…
Dr. William Lynes completed his residency in 1987 at Stanford University. He began his practice at the Kaiser Permanente Riverside Medical Group in California. It was the idyllic life that…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
BCMA is a protein that is found in multiple myeloma cancer cells. CAR-T cell therapies such as Carvytki are engineered to home in on BCMA proteins. According to a…
According to a recent article in the Pharma Times, E02463 is a therapy for indolent or slow growing, not yet problematic non-Hodgkin B cell lymphoma (iNHL). Enterome, its developer,…
According to a recent article in People Magazine, this Illinois family has a dream: to turn amyotrophic lateral sclerosis (ALS) from a fatal to a chronic illness in Brian’s…
If you’ve been there, then you know. Patients experiencing cancer, especially the rare aggressive types such as pancreatic cancer, and requiring targeted treatment for the disease may soon be…
The five-year rate of survival for pancreatic cancer is at 12% having the highest mortality rate of any major cancer. Defence, an aptly-named biopharmaceutical company in Vancouver, B.C. recently…
A recent article published in GlobeNewswire captured the results of the trial that was presented at the 2023 Annual ASCO Meeting from June 2nd to June 6th, 2023 in Chicago.…
At ASCO’s 2023 convention for patients diagnosed with glioma and a specific genetic alteration, Servier Pharmaceuticals announced that vorasidenib, the drug being investigated in its Phase III trial (INDIGO)…
The FDA advisory panel which is composed of specialists who advise the FDA on brain medications, recently voted 6-0 in agreement on that the new drug, Leqembi, was effective…
Patients who have been heavily pretreated for relapsed/refractory multiple myeloma participated in the phase 2 study of bispecific T cell redirection antibodies. The participants responded to treatment with a combination…
June 17th is International CDKL5 Day. During the month of June let’s tag every day with a reminder to those in the CDKL5 community to promote collaboration and fundraising.…
Effective June 1, 2023, the FDA lifted its clinical hold on Foghorn Therapeutics’ Phase 1 study of FHD-286 dose escalation in patients with acute myeloid leukemia and myelodysplastic syndromes. The…
An observational study of 599 survivors of COVID-19 was conducted at the Papa Giovanni Hospital in Bergamo, Italy three months after hospital admission. The patients were evaluated for thyroglobulin…
Relyvrio was officially approved by the FDA for treating ALS in September 2022. However, according to an article in BioPharma Dive, there was considerable controversy surrounding the clearance of the…
An announcement by Pfizer was recently published in Biospace outlining the results of BASIS its Phase 3 clinical trial (NCT03938792). The drug being studied, marstacimab, met its primary endpoints…
Céline Dion’s new milestone is her first-ever film, Love Again, which she stars in while living with a rare disease called stiff person syndrome. Céline expressed her wishes that…
A court in Zabbar, Malta found the parents of seven-year-old Victoria guilty of her death by negligence. The court also determined that the system had failed the child by…
In Japan researchers at the Juntendo University have discovered that when ECHS1 enzyme variations of mitochondrial enoyl-CoA hydratase short chain 1 (ECHS 1) do not function properly, they cause…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis. According to an article in BiopharmaDive, Ayvakit had been…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
Fierce Biotech recently carried an article about the death of Terry Horgan. Terry was a 27-year-old Duchenne muscular dystrophy (DMD) patient and the brother of the CEO of the…
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