Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

    New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease

    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…

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    Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune Diseases

      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…

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    An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

    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

    GAPVAC Project Tests An Individualized Form of  Immunotherapy to Treat Aggressive Brain Cancer

    A recent article in the journal Nature reports that Phase 1 GAPVAC-101 First-In-Human trial is one of the first clinical trials with a goal of designing and manufacturing actively personalized vaccines…

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    Secondary Long-Term Prophylaxis Shows Promise for Patients with Severe von Willebrand Disease (vWD)

    A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…

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    A Record Number of FDA Drug Approvals in 2018 Is Followed by an Encouraging Start for 2019

    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…

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    International Genetic Research Group to Begin Study of Hypermobile Ehlers-Danlos Syndrome (hEDS)

    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…

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    MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer
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    MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer

    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…

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    FDA Regulations and the Critical Differences Between Medical Device Trials and Pharmaceutical Trials

    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,…

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    Scientists are Ramping Up Their Attack on Alzheimer’s Disease Through Immunotherapy

    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…

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    Rare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis

    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…

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    Researchers Fail to Find a Cure for MPS Through Genome Editing

    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…

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