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.
An opinion expressed by Sheila Frame, President of Americas at Amryt Pharma, was published recently in the Pittsburgh Post-Gazette. Ms. Frame, who serves on the board of the Rare…
Little 2 1/2-year-old Raiden Pham cannot walk or talk, but he can smile at his parents and radiate love. The baby’s parents are on a 24/7 schedule caring for…
Jenny Decker’s disease, Charcot-Marie-Tooth (CMT), is named after the three doctors who first discovered the disease in 1886. CMT is a progressive neurological disease that affects approximately 2.6…
Jason Tolson age 30 of Yorkshire, UK had not been diagnosed when he first began to have symptoms in May 2022. Jason began to experience muscle and joint pain…
Genethon is a non-profit organization committed to the design and development of gene therapies for rare diseases. This unique company is a pioneer in identifying genes relating to genetic…
Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg…
Singer Celine Dion recently acknowledged that she has an extremely rare neurological disorder called stiff person syndrome (SPS). According to a recent article in The Metro, the disease occurs…
New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…
On January 6, 2023, the FDA granted conditional approval to a new Alzheimer’s drug, lecanemab, that will be sold as Leqembi. Vox carried the original story and included an…
Mazidul Islam told the Daily Star that he had to watch his 14-year-old son Farid die and could do nothing to save him. Among other things, he regrets the fact…
An antidepressant drug that was approved by the FDA in 1959 is being put to new and promising use to fight hard-to-treat breast cancers. The antidepressant, imipramine, was approved to…
Michael Myers, M.D., CEO of the Quoin pharmaceutical company describes Netherton Syndrome as a devastating skin disorder that at times may be fatal. In a recent article published in BioSpace,…
Cognitive deficits have been recognized as symptoms of advanced liver disease for many years. As reported in Bioengineer, the American Journal of Pathology recently published a study providing insights…
The 64th ASH Annual Meeting held on December 10, 2022, focused on various approaches for hematologic diseases with the goal of improving quality care and outcomes. Dr. Stephanie Lee, of…
Jenny Decker of Fenton, MO. interviewed with KMOV4 explaining that she was unable to walk when she was three years old due to a rare disease called Charcot-Marie-Tooth disease. Doctors…
Sangamo Therapeutics, with its primary focus on genomic medicine, recently issued a news release announcing data from its phase 1/2 STARR trial. The data evaluated isaralgagene civaparvovec, or (ST-920) as…
Rare disease stakeholders are called on to participate in the first Rare Disease Therapeutic Alliance of 2023 featuring an agenda presented by Rare Advocacy Movement experts. The agenda will…
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…
423 words 10% matched vs 524 words 5% matched The Rare Diseases International Organization reports that the groundbreaking UN Resolution on Persons Living With a Rare Disease (PLWRD), effective…
One year ago, in December 2021, clinical studies of lovo-cel for patients who were eighteen or younger were put on partial hold by the FDA. At the time, bluebird…
Continued From Part One Multiple myeloma is caused by the buildup of abnormal white blood cells that form tumors found primarily in bones. Available treatments work for short periods…
Globe Newswire recently published an article announcing positive results from Amolyt Pharma’s Phase IIa proof of concept clinical trial of AZP-3601. Amolyt Pharma, Cambridge, Massachusetts, and Lyon, France, is a…
Part 1 Newswise recently published exciting news of a therapy that causes the immune system to destroy cancer cells in the bone marrow. Researchers at Icahn School’s Tisch Cancer Center…
Waldenstrom macroglobulinemia (WM) is defined as a B-cell lymphoplasmacytic lymphoma. Data from patients in Latin America on WM are scarce. Researchers are hoping to fill the void by analyzing…
The latest statistics on heart disease and its effect on Black communities are startling. According to an article in the Dallas Examiner, Black people are fifty to seventy percent more…
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