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.
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…
John knew from an early age that he not only loved music but had confidence in his musical ability. However, there was another very troubling issue in his life. John…
The biotechnology company Anixa Biosciences, Inc. together with the Moffitt Cancer Center, issued a press release on May 22nd stating that the second patient had begun treatment as part…
Thirty-year-old Brendan O’Reilly was told that his rare disease affects only one person in 250,000. He was also told that he is lucky to be alive. Brendan’s symptoms confused…
The Chow family was told that their 33-year-old daughter, Brittney, would have to wait about twelve years for a kidney transplant. There were currently no kidneys available for transplantation. Elizabeth…
In March the FDA’s expert advisers voted 11 to 2 in favor of approving Roche’s antibody drug Polivy to treat lymphoma patients. According to an article in BiopharmaDive, the decision…
The safety, pharmacokinetics (absorption), and tolerability of the investigational eIF2B activator ABBV-CLS-7262 is being evaluated in a Phase 1b clinical study to treat patients with Vanishing White Matter (VWM) Disease.…
PhRMA Public Affairs recently carried the good news that Governor Holcomb of Indiana has joined the governors of Arkansas and West Virginia by signing a law aptly named 'Share the…
This week PR Newswire carried an announcement by the biotechnology company Immunic Inc. of preclinical data confirming that Vidofludimus Calcium (IMU 838) may be a Nurr 1-Activator reinforcement. Immunic is…
For patients who were given a diagnosis of advanced, inoperable (unresectable) biliary tract cancer, an article appeared in Healio with some encouraging news. Gemcitabine and cisplatin drugs have…
Omisirge (omidubicel) is the first allogeneic (from a donor) product for SCT that has been the recipient of FDA approval. The FDA's nod moves Omisirge into the realm of…
The damage caused by cocaine can sometimes be misdiagnosed as a rare disease: granulomatosis with polyangiitis. Cocaine use has been increasing in America and the UK, resulting in serious…
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…
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