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 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.…
Continue ReadingPositive Results in Cancer Vaccine Trial for Merkel Cell Carcinoma and Cutaneous Squamous Cell Carcinoma
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)…
Continue ReadingASCO 2023: Vorasidenib Hailed As “Unprecedented Improvement” in Glioma Treatment
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…
Continue ReadingFDA Panel Backs Approval of a New Drug for Alzheimer’s
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…
Continue ReadingBispecific T-Cell Redirection Antibodies in the Treatment of Multiple Myeloma
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…
Continue ReadingFDA Lifts Clinical Hold on Phase 1 AML and MDS Clinical Trial
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…
Continue ReadingCould COVID-19 be a Cause of Autoimmune Thyroid Disease?
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…
Continue ReadingALS Drug Facing Possible Rejection by the European Medicines Agency
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…
Continue ReadingExperimental Therapy for Hemophilia Meets Phase 3 Trial 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…
Continue ReadingCeline Dion Isn’t Slowing Down Despite Stiff Person Syndrome Diagnosis
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…
Continue ReadingParents in Malta Handed a Prison Sentence for the Death of Daughter Who Died of Aplastic Anemia
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…
Continue ReadingA High-Efficiency Assay with Rapid Mitochondrial Disease Diagnosis
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…
Continue ReadingThe FDA Has Approved Ayvakit for the Treatment of Indolent Systemic Mastocytosis
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…
Continue ReadingRNA Sequencing: Hope for Improved Rare Disease Diagnosis
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…
Continue ReadingDuchenne Muscular Dystrophy Patient Dies in Gene Therapy Trial
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…
Continue ReadingThis Spinal Muscular Atrophy Patient Established a Nonprofit for Disabled Artists
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…
Continue ReadingOvarian Cancer: Second Patient Treated with Novel CAR-T in Trial
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…
Continue ReadingAcute Disseminated Encephalomyelitis Can be Confused for a Stroke
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…
Continue ReadingThere Is No Limit to What Some Mothers Will Do For Their Children.
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…
Continue ReadingThe FDA Approves Roche’s Antibody Drug Polivy to Treat Lymphoma Patients
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.…
Continue ReadingABBV CLS 7262 is Being Evaluated as a Potential Treatment for Vanishing White Matter Disease in the Brain
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…
Continue ReadingIndiana Legislature Passes Bill Protecting Patients from Paying More Than Their Insurers
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…
Continue ReadingImmunic Inc. Announces Promising Pre-Clinical Findings for Multiple Sclerosis
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…
Continue ReadingA Glimmer of Hope for Patients with Advanced Biliary Tract Cancer
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