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 posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched…
Continue Reading
The LLS Ground-Breaking Precision Medicine Approach Master Trial BEAT AML Releases Third Year Test Results
In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…
Continue Reading
Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance
Dr. David Porteous, a General Practitioner in Bristol, England recently reported to the Bristol Cable, a community-run newspaper, that he does not agree with the extensive list of treatments being…
Continue Reading
GPs and Specialists in UK are Challenging the Restrictions That Deny Patients Access to Treatment
Ash Clinical News recently interviewed Dr. Bruce Leff of Johns Hopkins in connection with “hospital-at-home”, one of the major changes occurring in hospital care. Dr. Leff and his team at…
Continue Reading
In-Patient Hospitalization vs. At-Home Programs: Will Doctors Be Making House Calls Again?
According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…
Continue Reading
New Drug Under Review by the FDA to Treat the Most Lethal Form of Spinal Muscular Atrophy
Just one year ago David Chen, a forty-year-old policeman who lives in Toronto, was walking home after playing volleyball. According to a recent article in CBC Canada News, a…
Continue Reading
The FDA Rejects a Therapeutic Device that Stimulates Cranial Nerves
Although guidelines for Parkinson’s patients suggest that they visit a specialist every six months, according to a report in Parkinson’s News Today, the waiting period may be up to…
Continue Reading
New Personal Kinetigraph (PKG) Allows Parkinson’s Patients in the UK to Connect with their Specialists
Lithium boosts muscle strength in mice with rare muscular dystrophy New drug target identified Click to share on Pinterest (Opens in new window) Removing one gene caused normal muscle muscle…
Continue Reading
Lithium boosts muscle strength in mice with rare muscular dystrophy
The mesothelioma diagnosis was a surprise to Raya Bodnarchuk of Baltimore, Maryland because she had never worked in a factory, mine, the military, or a shipyard. These are the typical…
Continue Reading
First Bronchitis, Then Pneumonia, Then the Stunning Diagnosis: Mesothelioma
A birth involving mitochondrial replacement therapy took place as part of a clinical trial in Greece according to a recent article in STAT. Greece is now the second country…
Continue Reading
Can the Mitochondrial Replacement Therapy Ban in the U.S. Be Lifted?
Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…
Continue Reading
Artificial Intelligence(AI) and a Solution to Detect Familial Hypercholesterolemia
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
Continue Reading
A Team of Scientists Researching A Cure for Neurofibromatosis Type 1
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
Continue Reading
Announcing A New Test for A Wide Range of Rare Metabolic Disorders
An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…
Continue Reading
New England Journal of Medicine (NEJM) Announces Results of A Unique Study of Hirschsprung Disease Affecting Infants
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
Continue Reading
The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation
An article in Austin Gastroenterology recently asks this question. It also confirmed what we already know, that genetic testing is now available to the average person and in fact,…
Continue Reading
Can We Entrust Our Health to Genetic Mail-Order Tests?
LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug…
Continue Reading
uniQure Receives FDA Fast Track Designation for AMT 130 Gene Therapy for the Treatment of Huntingtons Disease
An encouraging article recently published in Sickle Cell Anemia News announced that due to the urging of pharmaceutical companies and patient advocacy groups, the FDA has produced an updated draft…
Continue Reading
New, Revised FDA Guidelines for Rare Diseases: Common Issues in Drug Development
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the FDA based on interim results of the clinical trial ADMIRAL, according to an article in EurekAlert for Science News. A…
Continue Reading
FDA Approved Gilteritinib May Increase Cure Rates for Mutated FLT3 Genes in AML
OncLive recently published the views of several speakers at its State of the Science Summit on breast cancer. Stephanie LaBomascus, MD gave a presentation that highlighted triple negative breast…
Continue Reading
The Latest Advances in Triple Negative Breast Cancer
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the USFDA based on interim results of the clinical trial ADMIRAL according to an article in EurekAlert for Science News. A total of 371 adult patients…
Continue Reading
Targeted drug for leukemia tested at Penn Medicine helps patients live longer
A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
Continue Reading
New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease
A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
Continue Reading
A Rare Intervention For His Children’s Alkaptonuria
According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited. The second finding…
Continue Reading
Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune Diseases
Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
Continue Reading
An International Organization Dedicated to Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals