About five months ago, Lexi Robins was born in the United Kingdom. She didn't show any signs of abnormal development at first, besides the fact that her thumb didn't move…
According to a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…
As reported in Times Union; this was a year where the numbers of many contagious diseases saw steep declines as the world stepped up in handwashing and mask-wearing. In the…
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…
What do phenylketonuria (PKU), galactosemia, and maple syrup urine disease (MSUD) have in common? All three are considered inborn errors of metabolism, or rare genetic disorders in which the…
In a review posted on dovepress.com, monoclonal antibodies have established themselves as an important tool in the management of multiple sclerosis in both the relapsing-remitting form and the progressive form.…
Ally Winter was diagnosed with short bowel syndrome when she was four years old. Now, she is 17 years old and working to raise awareness for rare and chronic illnesses.…
Happy Friday! This week, we examine the struggle to access experimental rare therapies through compassionate use. Also, in our new Compassion Corner series, we examine why addressing patients' personal wellbeing can…
Currently, there are few - if any - ways to treat patients with myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS). Rather than treating the condition itself, doctors often treat symptoms…
The Hindustan Times recently published an article that again puts CRISPR at the forefront of medical news. The newest CRISPR tool was designed to identify Sars-CoV-2 and prevent the transmission…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) recently held the latest webinar in its Global Education series on July 9, 2021. This webinar was a summary of research presented at the…
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…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
Have you ever heard of isoflavone? Well, isoflavone is part of a class of phytoestrogens, or plant-based compounds which resemble estrogen. According to Medical XPress, research from Dr. Ashutosh Mangalam,…
Breakthrough Therapy designation is granted by the FDA as a way to accelerate drug development and review. In particular, the designation is granted to drugs or biologics for serious conditions,…
According to an article in NCBI’s Drug In Context, it took the combined services of the internal medicine, neurology, and psychiatry departments to diagnose this rare disease. Anti-NMDAR encephalitis…
On July 14, 2021, biopharmaceutical company Alkeus Pharmaceuticals, Inc. ("Alkeus") shared via press release that its therapy ALK-001 (C20-D3-vitamin A) received Breakthrough Therapy designation from the FDA for the treatment…
Spinal muscular atrophy is rare progressive muscular disease that over time, erodes a patient’s movement. Without early intervention, the condition erodes the young patients ability to breathe and even swallow.…
Medical professionals are still working to uncover the cause of Alzheimer's disease. While they believe that a combination of genetic and environmental factors paired with certain lifestyle choices leads to…
According to a story from Parkinson's News Today, a three-year study has determined that dancing to music weekly may help slow the progression of Parkinson's disease symptoms. This effect included…
Normally, B lymphocytes ("B cells") are a type of white blood cell that plays a role in immune function. In healthy individuals, B cells create antibodies to fight infections and…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
In a news release from mid-July, 2021, biopharmaceutical company Strongbridge Biopharma plc ("Strongbridge") shared the publication of new post hoc analyses regarding KEVEYIS (dichlorphenamide) for patients with Primary Periodic Paralysis…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
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