Why Rare Diseases Pose a Diagnostic Challenge As discussed in the Orphanet Journal of Rare Disease, a "rare disease" is defined as a condition affecting fewer than 1 in 2,000…
Authored by Dr. Harsha Rajasimha, Founder and Executive Chairman, IndoUSrare. Co-Authored by Dr. Padma Rammoorthy, Medical Consultant, IndoUSrare. The diagnostic journey for patients with rare diseases often becomes a prolonged odyssey,…
Often when a cure for a specific rare disease has yet to be developed, patients have enrolled in clinical trials with positive results. However, FDA-approved trials have developed a reputation…
Nancy Yu co-founded AllStripes with a vision in mind: to unlock more treatment options for those with rare diseases. In Part 1 of our interview, Patient Worthy sat down with…
Disruption in clinical trial procedures can be added to the list of changes that have occurred since the start of the pandemic in 2020. The clinical trial sites had always…
Dr. Duneel Dhand, an internal medicine physician and author, told KevinMD about two events in his life that motivated him to treat his patients with compassion. A Doctor’s Character As…
Gene therapy is a recent development in the industry, and it is one that has brought hope to many people who live with a genetic condition without a viable treatment…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
Over the past decade, scientists have made many life-saving advances in all areas of medicine. They have created molecular agents to target neurodegenerative diseases. But they were stopped right there…
Balance issues are often a difficult symptom to detect and track in those with multiple sclerosis. It is important that these problems are addressed by doctors, as they can lead…
Research has recently been published in Nature Communications that shows the success of a new gene therapy in the treatment of Huntington's Disease. This therapy, an adeno-associated virus-based gene therapy, was tested…
The validity of Morgellons disease, which has been associated with Lyme disease, has been widely debated for years. Those affected by it swear that it is real, that fibers grow…
The Patient Seminar Series is an event in which those who are affected by rare diseases in some way share their experiences. Patients, family members, medical professionals, and legislators all…
Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…
Dr. Halima Moncreiffe is an assistant professor at Cincinnati Children's Hospital Medical Center, and she focuses on autoimmune diseases. She recently participated in an interview with Front Line Genomics, in…
Young-onset Parkinson's disease is a mystery to medical professionals. The onset of this condition typically occurs in those older than 60, but in this form the onset occurs between age…
ALS is a progressive disease that causes the loss of muscle function, and it often develops very quickly after a diagnosis. Rami Epstein, the CEO of the biopharmaceutical company called…
New knowledge of the genes that affect sickle cell disease and beta thalassemia has allowed for advances in treatments for these diseases. Advances in technology have also helped with…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
There have been extreme improvements in genetic engineering over the past fifty years since it was pioneered in the 1970s. It has been used for the treatment of various…
For a long time, Huntington's Disease has been monitored and studied through the examination of subjective measures, such as Unified Huntington Disease Rating Scale (UHDRS), and survey-based questions. MC10 is…
Patients in the ICU are under constant surveillance, with consistent monitoring of their blood pressure, blood cultures, and other laboratory markers. These things help doctors understand how the patient is…
Rare diseases often lack a voice. Unlike more common diseases, they do not have big movements that bring the disease awareness through fundraisers, 5Ks, and spokespeople. However, sometimes, celebrities have…
In an article posted by the Wyoming Medical Center, Dr. Adam Linck clears up some common questions concerning hereditary hemochromatosis. People living with this condition have an excess amount of…
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