Firdapse, or amifampridine phosphate, is a medication currently approved for Lambert-Eaton myasthenic syndrome, or LEMS. However, Catalyst Pharmaceuticals has been working to evaluate if it can aid other neurological and…
According to a story from Targeted Oncology, the US Food and Drug Administration (FDA) has granted Fast Track designation to the experimental drug OMP-305B83, also known as navicixiumab. This designation…
According to a story from pharmanewsdaily.com, the gene therapy company Avrobio has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AVR-RD-02, an…
According to a story from tmcnet.com, Alkermes plc and Biogen, Inc. have recently announced that the US Food and Drug Administration (FDA) has approved a new treatment for multiple sclerosis.…
According to a story from NBC News, the US Food and Drug Administration (FDA) issued an announcement during the summer detailing a patient death linked to the use of fecal…
According to a story from statnews.com, the approval of the new cystic fibrosis drug Trikafta has caused quite a buzz in this rare disease community. While this new drug offers…
"We went from being able to treat no patients to being able to treat nearly all patients in seven years." 90% of cystic fibrosis (CF) patients now have a new treatment…
According to a story from The Washington Post, data regarding the recently approved drug Trikafta, which is designed to treat cystic fibrosis, was so convincing that the US Food and…
FA Fanconi anemia (FA) is a rare disease in which the bone marrow in the body essentially fails. It is a genetic condition where the body progressively becomes deficient in…
According to a story from newswise.com, biologic medicines have had a major positive impact on outcomes for patients with the rare disease juvenile idiopathic arthritis (JIA). However, as these medicines…
GlaxoSmithKline (GSK) has just announced that Zejula, otherwise known as niraparib, has just been approved for cancer patients beyond those who have a BRCA+ mutation. The patients now included in…
According to a story from Buzzfeed News, Facebook has been receiving more negative press than ever lately, and so far it often seems justified. From the Cambridge Analytica scandal to…
According to a story from the Times of Israel, a team of researchers associated with the University of Massachusetts appear to have discovered a potential breakthrough in regards to the…
According to a story from BioSpace, the biopharmaceutical company Newron Pharmaceuticals S.p.A. has announced that is partnering with the Rett syndrome disease community in order to sponsor an all new…
Narcolepsy is a rare neurological disease that affects the sleep cycle. Symptoms include sleep paralysis, excessive daytime sleepiness, disrupted nighttime sleep, hypnagogic hallucinations, and, for around 70% of patients, cataplexy.…
According to a story from BioSpace, Chugai Pharmaceutical Co., Ltd. has recently announced that the Marketing Authorisation Application for the its drug satralizumab has been accepted by the European Medicines…
The number of systemic juvenile idiopathic arthritis patients who develop lung disease (sJIA-LD) is rising in both the U.S., Europe, the Middle East, and Canada. This trend has been notable…
That’s right- HOBSCOTCH NOT hopscotch, and you don’t need a sidewalk and chalk to play it. The Epilepsy Foundation of Virginia (EFVA) has been looking out for you, and applied…
Rest In Peace, Senator Kay Hagan, who unexpectedly died Monday at the age of 66. It has been widely reported that Senator Hagan was diagnosed with encephalitis in 2016, which…
Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…
The Gap Tysabri is a therapy that has shown efficacy for reducing disease activity in patients diagnosed with pediatric-onset multiple sclerosis (POMS). Previous investigations have indicated that up to 58%…
Niemann-Pick disease type C (NPC) is a rare disease caused by a defected NPC protein. In a healthy individual, this protein helps the body process cholesterol. Those diagnosed with NPC experience…
According to a story from MDMag, the US Food and Drug Administration (FDA) has recently approved a label expansion for the drug treprostinil (marketed as Orentiram). This update to the…
According to a story from irishmirror.ie, Danny O'Donoghue, the vocalist and keyboard player for the Irish rock band The Script, recently talked publicly about his struggle with Wilson's disease, a…
Teresa Scruton is not a physican nor a nutritionist but she has been a person with hereditary angioedema (HAE) as well as a researcher for many years. Below she shares…
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