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.
Over the years, there has been minimal improvement in the life expectancy differences between white and black people living in the United States. Yet, according to an article featured…
Continue ReadingA New National Analysis Indicates that Higher Levels of Black Primary Care Physicians Means Longer Life Expectancy for Black Patients
The FDA has carried on a year-long disagreement with BrainStorm Cell Therapies, the developer of the experimental stem cell treatment called NurOwn. It is referred to as a personalized cell…
Continue ReadingBrainStorm Files an ALS Drug Application Despite the FDA’s Protest
On March 22, 2023, via PRNewswire, the FDA announced that its CNS Drugs Advisory Committee voted unanimously in favor of a potential accelerated approval of tofersen to treat people with…
Continue ReadingAmyotrophic Lateral Sclerosis Drug Developers Set Their Sights on a New Treatment Target
No One Should Face Cancer Alone. That is the mission of the non-profit Cancer Support Community (CSC). It offers emotional support and hope via programs designed for cancer patients, cancer…
Continue ReadingFor Patients Under Consideration for CAR-T Therapy The Cancer Support Community/Gilda’s Club Has A Free Program
In 2003 Ricky Safer was diagnosed with primary sclerosing cholangitis (PSC). Her doctors were not able to tell her much about the origin of her disease. Nor were they able…
Continue ReadingThe Chan Zuckerberg Initiative is Providing Financial Support for Patient-Centered Rare Disease Research
Theravance Biopharma’s late-stage failure suddenly became a jump-off point for treating multiple system atrophy (MSA) according to a recently published article in Neurology Live. After the failure of two…
Continue ReadingAmpreloxetine Failed the First Time but Emerged a Winner for Multiple System Atrophy
Caring is Sharing and Getting Involved. On April 17th Landmarks all over the world will be asked to ‘Light It Red’ in honor of Hemophilia Day. The theme of this…
Discovering a treatment for a rare disease is challenging but it can also be rewarding. This is especially true when treating an extremely rare disease such as perivascular epithelioid…
Continue ReadingFDA Approves New Treatment for Rare Cancer PEComa
Ayla Bashir and her parents, Sobia Qureshi and Zahid Bashir, have participated in a medical first with Ayla surviving Pompe disease. The same disease caused the death of her…
The Ovarian Cancer Research Alliance stated in new guidance that evidence indicates the majority of ovarian cancers begin in fallopian tubes. The president of the alliance, Audra Moran, was…
Continue ReadingA Research Group Finds Most Ovarian Cancers Begin in the Fallopian Tubes. Should They Be Removed?
The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…
Continue ReadingMutual Trust Between Doctors and Parents of Children with Rare Diseases Means Optimum Care for the Child
Dr. Amish Desai speaks with the utmost sincerity on KevinMD's recent podcast. The doctor explained the difficulty he encountered trying to balance treatment for his father while giving the same quality…
Continue ReadingA Doctor’s Struggle to Give Optimum Care to His Father and Patients
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…
Continue ReadingCompassion Corner: How to Avoid Compassion Fatigue in Nursing
In March of 2021, twenty-eight-year-old Alix Burnard of Newbury, Berkshire UK had several Covid-19 tests due to a suspected infection. All three tests were negative, so she felt it…
Continue Reading28-Year-Old Alix Lived a Full Life in the Face ALK Positive Lung Cancer
Imagine being Rowan Marshall, a seven-year-old first-grade student with a debilitating rare disease. Rowan made it through the new student formalities. He is doing well at school. Now he faces…
Continue ReadingParents of a Boy with a Charcot-Marie-Tooth Disease are Asking His School for Help
Researchers have been grappling for years with the STAT5 protein as a tool to fight cancer. STAT5 is critical because it is partly responsible for the development and progression…
Continue ReadingResearchers have Discovered a Method for Targeting a Previously “Undruggable” Protein in Cancer
After winning the 1998 100m butterfly in Perth, breaking a world record, winning 54 international medals, and becoming the Swimming Hall of Fame inductee in 2022, Michael Klim appeared…
Continue ReadingBeing an Olympic Swimming Legend Doesn’t Ease the Burden of Chronic Inflammatory Demyelinating Polyneuropathy
In June 2022, Andrew Mace received CPR from a neighbor, 21-year-old Alex Duncan, a med student who luckily remembered her instructions while at Cambridge University, UK. Andrew, age 44,…
Continue ReadingThis Father Collapsed in Front of his Kids Because of Fabry Disease
The failure rate of drug development for Alzheimer’s is 99 percent according to a recent study. Since 1984 scientists have concentrated on developing treatments to prevent the formation of…
Continue ReadingAlzheimer’s Disease: An Influential 2006 Study Containing False Images Leads Scientists To Reverse Previous Theories
Emergency Room (ER) doctors are gradually being replaced by physician assistants and nurse practitioners. The Neiman Institute reported that between 2005 and 2020 the number of ER visits with midlevel…
Continue ReadingThe Journal of Emergency Medicine: By 2030 The Number of Midlevel Practitioners Will Equal the Number of Doctors In the ER
A town hall meeting was held on February 7th of this year to discuss designs for clinical trial gene therapies. According to a report in Pharmaceutical-Technology, the FDA experts were…
Continue ReadingClinical Development of Gene Therapy Products for Rare Diseases
This week the family of actor Bruce Willis announced that he received a diagnosis of frontotemporal dementia (FTD), a rare disease that causes behavioral changes, language, speech, and memory…
Continue ReadingThe Family of Actor Bruce Willis Acknowledges his Diagnosis of Primary Progressive Aphasia
509 words 8% matched vs 786 words 5% matched A new study recently appeared in the publication The Conversation written by authors Laura Whitworth, Group Laboratory Manager, and Prof. Lalita…
Continue ReadingGaucher Disease Appears to Protect Against Tuberculosis in Ashkenazi Jews
Researchers are closing in on cancer from every angle. Recently a team at the Colorado University (CU) Cancer Center has been focusing on p53, a common gene mutation found…
Continue ReadingResearchers Discover Mechanism for Triggering the Death of Cancer Cells
Stat News recently published comments by Bruce Bloom, collaboration officer at Healx, Cambridge, UK heralding the major advances in the treatment and the study of rare disease during 2022.…
Continue ReadingThe Number of Rare Diseases has Risen to 10,867, Affecting over 300 Million People
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