Many of us are familiar with the famous story written by Roald Dahl titled Charlie and the Chocolate Factory; we know the tale of the golden ticket and Charlie's journey to…
Ashley O’Keefe is a proud mother of three and a nurse, so she's seen her fair share of children and health problems. So when her third child was born, she…
According to a recent press release, the Australian Human Research Ethics Committee (HREC) has granted approval to Claritas Pharmaceuticals' submission for R-107. Now, the company is free to move forward with…
According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone - marketed as ZTALMY - as a…
Ovarian cancer is notoriously hard to treat, ranking as the fifth leading cause of cancer deaths in women. Diagnosis often does not come until the disease has already progressed, making…
According to a recent press release from Respira Therapeutics, their investigational treatment for pulmonary arterial hypertension (PAH), RT234, has just been administered to the first patient in a phase 2b…
The American Association for Cancer Research (AACR) Annual Meeting took place from April 8th until the 13th, with lots of interesting data and innovations presented by some of the leaders…
One of the largest problems that stands in the way of rare disease research is funding. It can be difficult to raise the enormous amounts of money needed to plan,…
According to a recent article from WFMZ+, the FDA has recently approved Regeneron's supplemental Biologics License Application (sBLA) for Dupixent, granting it priority review. Dupixent, also known by the generic…
According to a recent press release, the FDA has recently granted approval to Vijoice - also known under the generic name alpelisib - for the treatment of PIK3CA-related overgrowth spectrum…
A recent study titled "Effect of FT218, a Once-Nightly Sodium Oxybate Formulation, on Disrupted Nighttime Sleep in Patients With Narcolepsy: Results From the Randomized Phase 3 REST-ON Trial," was published…
Adverse events (AEs) are unexpected medical issues that arise upon treatment. Now, this is a very broad term, and it can be broken down into more specific categories depending on…
The American Academy of Neurology (AAN) 2022 Annual Meeting is held from April 2nd until the 7th in Seattle, with its virtual counterpart happening on April 24th to the 26th.…
The rarity of certain diseases often stops them from having numerous treatment options, and the variability of patient experiences makes it so that each option does not necessarily work for…
Celia Grace, a metachromatic leukodystrophy (MLD) patient from Alabama, has recently made history in Minnesota - and she's just five years old! Celia recently underwent an investigational gene therapy to…
Cushing's disease, like all other rare diseases, benefits greatly from increased awareness and advocacy. Because there are fewer patients and affected individuals, their voices need to be louder in order…
Recent research has given us a better understanding of acromegaly, a rare hormonal disorder. Conducted by Maria Fleseriu, MD, FACE, and colleagues at the Pituitary Center at Oregon Health &…
CANbridge Pharmaceuticals recently announced via press release that the Taiwan Food and Drug Administration (TFDA) has accepted their New Drug Application (NDA)/Orphan Drug Registration (ODR) for CAN108. Marketed as LIVMARLI,…
Awareness and advocacy are extremely important to every rare disease, and porphyria is no different. That's why we celebrate Porphyria Awareness Week (PAW) every year from April 2nd until the 9th.…
According to a recent press release from Passage Bio, their investigational gene therapy for Krabbe disease, PBKR03, may be ready to move into the clinical trial stage of drug development.…
Bristol Myers Squibb (BMS) has been developing luspatercept-aamt - marketed as Reblozyl - as a treatment for anemia in non-transfusion dependent (NTD) beta thalassemia. They submitted a supplemental biologics license…
In order to create treatments and diagnostic methods for rare diseases, it's necessary to have a thorough understanding of said disease. A recent study aimed to do just this. Titled…
We often think of cyclic vomiting syndrome (CVS) as a rare disease, but is this really the truth? According to a recent article from New Castle News, this is not…
Pancreatic cancer is typically a difficult cancer to treat, leading to poor outcomes and high mortality rates. One of the reasons for this is the difficulty of diagnosis; it often…
Sparsentan is a dual endothelin angiotensin receptor antagonist (DEARA) currently being developed for the treatment of IgA nephropathy (IgAN). The newest update on sparsentan was recently shared by Travere Therapeutics via…
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