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 in Parkinson’s News Today reports that a reduction in a patient’s required medication and management of motor system disorders may be achievable with Deep-Brain Stimulation (DBS) for…
Continue ReadingParkinson’s Patients May Benefit from Deep-Brain Stimulation (DBS)
Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…
Continue ReadingA Record Number of FDA Drug Approvals in 2018 Is Followed by an Encouraging Start for 2019
A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…
Continue ReadingInternational Genetic Research Group to Begin Study of Hypermobile Ehlers-Danlos Syndrome (hEDS)
According to a recent article in the Globe, the Danish biotech company Saniona AB has initiated a Phase 2a clinical trial of Tesomet for the treatment of hypothalamic obesity, a rare…
Continue ReadingA New Drug With the Potential to Treat Hypothalamic Obesity
A recent article published in online CANCER NETWORK reports findings that indicate children who have survived Hodgkin lymphoma (HL) may develop solid tumors in later years. These findings will have…
Continue ReadingFor Childhood Survivors of Hodgkin Lymphoma, the Battle may not be Over
Although some ultrasound and/or CT scan studies may show that patients’ lymph nodes (glands) are free of disease, upon further examination using sentinel lymph node biopsies,(SLNB) the doctors at MD…
Continue ReadingMD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer
Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…
Continue ReadingFDA Regulations and the Critical Differences Between Medical Device Trials and Pharmaceutical Trials
According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of…
Continue ReadingThis Dedicated Scientist Left a Legacy to Fight Cystinosis
In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…
Continue ReadingScientists are Ramping Up Their Attack on Alzheimer’s Disease Through Immunotherapy
Most biological drugs, including insulin, can't be taken orally, because enzymes and proteins cause the drugs to break down too quickly in the digestive system. For decades, the pharma industry…
Continue ReadingFirst Human Study of ‘Robotic’ RaniPill Designed to Replace Injections of Biological Drugs
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
Continue ReadingRare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis
Massimo Federico, MD, director of the medical oncology unit at the University of Modena and Reggio Emilia, Italy, was quoted in Cancer Network as saying that “Achieving a complete clinical and…
Continue ReadingStudy Shows Impressive Results Using 3 Drug Combo in High-Risk Follicular Lymphoma
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
Continue ReadingResearchers Fail to Find a Cure for MPS Through Genome Editing
A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…
Continue ReadingVancouver Researchers Find That Blood Cells Could Help Screen Ambroxol’s Efficacy in GD Type 3
Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…
Continue ReadingAlexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients
Ofatumumab as a Single Agent Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…
Continue ReadingResearchers Find the Future of Ofatumumab in Treating Follicular Lymphoma is Questionable
The treatment of metastatic renal cell carcinoma (RCC) has evolved over the last ten years and investigators now look towards combination therapies to improve response rates and durability for…
Continue ReadingMerck’s Keytruda in Combination with Pfizer’s Inlyta Significantly Improved Overall Survival and Progression-Free Survival in Therapy for Metastatic or Advanced Renal Cell Carcinoma
The Food and Drug Administration (FDA) recently announced its approval of Venetoclax (Venclexta) in combination with either decitabine or azacitidine (hypomethylating agents) or cytarabine (an antimetabolite) to treat adults 75 years…
Continue ReadingFDA Approves Venetoclax Combination For Newly Diagnosed AML in Adults 75 Years or Older
“Doctor” Onelio Hipolit-Gonzalez has been charged with unlicensed practice of a health care profession and unlawful use of a two-way communication device according to an article published in the Spectrum News of…
Continue Reading73-Year-Old Arrested by Undercover Agents for Posing as a Physician
A report published in 2018 in The Hemotologist mentions the fact that most of the therapies introduced over the years for acute myeloid leukemia (AML) simply meant optimizing the use of…
Continue ReadingFDA Approves Four Novel Therapies for Acute Myeloid Leukemia
The University of Louisville’s publication UL News recently reported a finding by its research team regarding an immune checkpoint molecule. The molecule had been developed for cancer immunotherapy but when…
Continue ReadingUniversity of Louisville’s Research Team Discovers Molecule That Protects Against Development of Cancer Cells
A recent interview with David Alvarez and his mother in the CTPost explains how he manages to remain positive after being diagnosed with spinal muscular atrophy (SMA). David is a freshman…
Continue ReadingCollege Freshman With Spinal Muscular Atrophy has Exciting Plans for the Future
In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58. Twelve patients were enrolled as…
Continue ReadingA Clinical Trial Now Testing a Novel, Engineered Enzyme for Patients with Classical Homocystinuria
A recent article in CheckOrphan reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the synthetic GLP-2 analog apraglutide for the treatment of short bowel syndrome…
Continue ReadingOrphan Drug Designation Granted to Therachon’s Drug Apraglutide by FDA for Treatment of Short Bowel Syndrome
ANCA Vasculitis News recently reported findings published in the journal Arthritis and Reumatology on the study “Efficacy and Safety of Belimumab and Azathioprine for Maintenance of Remission in ANCA‐Associated Vasculitis: A…
Continue ReadingBelimumab Maintenance Did Not Delay Relapse in AAV Patients
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