Lisa Stockmann Mauriello began her petition to Biogen almost one year ago. In her letter to the developers, Biogen and Ionis Pharmaceuticals, Lisa explained that she has an inherited form…
When Ashley and Connor Henderson received news that they were pregnant, the family was overjoyed. After all, the Henderson family already had Liam and was hoping to add another child…
When I was a teenager, my mom tried to convince me to become a nurse. As a nurse herself, she told me about the rewards of helping people, the challenges…
The month of August is recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This is a time of year in which the community works hard to spread awareness about this…
In an August 3rd news release, biopharmaceutical company RELIEF THERAPEUTICS Holding AG ("Relief") shared that its therapy RLF-100 (aviptadil) for sarcoidosis received Orphan Drug designation from the FDA. This…
Written by Dawn Laney, MS, CDC, CCRC The journey to a Fabry disease diagnosis is rarely a clear, straight path. Fabry can mimic more common health issues and lead patients…
The FDA has recently granted ONCOFID®-P the Orphan Drug designation for the treatment of malignant mesothelioma (MM). This designation is reserved for therapies indicated for rare diseases, and it provides…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
For many patients, certain treatments can be expensive, invasive, or difficult to access. But it doesn't have to be this way. According to aHUS News, researchers are running two separate…
Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
As many people know, writing a book is a huge accomplishment - and one that many people wish to achieve throughout their lifetime. For Samantha VanAlstyne ("Sam"), a 25-year-old who initially…
A study found on Medscape confirms that the human immune system and the human brain are the most complicated in our biological systems. The two systems are responsible for…
Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…
HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma patients haven't seen a drug approval for their disease state in a decade. That is until the FDA approved Keytruda in combination with…
Patients with relapsed or refractory mantle cell lymphoma (R/R MCL) may have a new treatment option, based on positive results from a Phase 1/2 trial of Zanubrutinib published in Blood…
On July 30, 2021, biopharmaceutical company Cyclo Therapeutics shared that it had released new and promising data regarding the safety, efficacy, and tolerability of Trappsol® Cyclo™ ("Trappsol Cyclo") for patients with…
According to a fairly recent news release from biopharmaceutical company Epygenix Therapeutics, Inc. ("Epygenix"), the company's therapy, EPX-200, received Orphan Drug designation from the FDA. Altogether, the therapy is designed…
According to a story from Pharma Times, the drug company Bristol Myers Squibb has recently announced that its therapy nivolumab (marketed as Opdivo) has been approved by the European Commission…
Earlier this year, Patient Worthy reported on a deadly cholera outbreak which claimed 20 lives the Bomadi and Burutu areas of the state of Delta within Nigeria. Unfortunately, continued struggles,…
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…
Over the last few months, researchers have been enrolling and dosing patients with hereditary angioedema (HAE) from Europe, Israel, and Canada within the Phase 2 RAPIDe-1 clinical trial. According to…
In case you missed it - a news release from biopharmaceutical company Pharnext SA ("Pharnext") shared that on July 12, 2021, the first European patient enrolled in the Phase 3…
According to an article published in EuroNews, the National Cancer Institute has announced that MIT researchers have developed a device that can locate cancer cells during urine tests. The device…
When Yang Siyang learned his eight-year-old Lillian has the rare childhood cancer acute myeloid leukemia, he felt overwhelmed by shock, grief, uncertainty. There are many intense and complicated emotions that…
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