The Orphan Drug designation was created to aid and hasten the development of drugs created for the treatment, diagnosis, or prevention of rare diseases. When the FDA grants this designation,…
When it comes to cancer treatment, it's important to understand what leads to improvements in survival; knowing these things helps to provide the best outcome for the patient. That's why…
According to a recent news release from PR Newswire, the European Medicines Agency (EMA) has recently awarded elamipretide its Orphan Drug designation for the treatment of Barth syndrome. This treatment,…
A recent study - which you can find in the journal Muscle & Nerve - investigated Soliris as a treatment for anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG). It…
Clinical trials are useful not only for initially establishing the efficacy of treatments for rare diseases, but for demonstrating that these results are durable as well. The MPOWER trial, which…
Understanding how a patient's condition will progress is extremely important in providing them with the best treatment, and autosomal dominant polycystic kidney disease (ADPKD) does not stray from this trend.…
Within the United Kingdom, there's a program titled the Innovative Licensing and Access Pathway (ILAP), which is meant to give patients earlier access to developing treatments. The first step to…
Getting a rare disease diagnosis can be difficult and take a long time. This can be a problem, as time is something that those with progressive illnesses don't have much…
According to a recent press release from Biospace, the first cohort of the ReSPECT-LM trial has officially completed enrollment. This trial focuses on Rhenium-186 NanoLiposome (186RNL) as a treatment for…
Medscape has recently reported on a new supplemental approval from the FDA for setmelanotide, which is a treatment for obesity associated with Bardet-Biedl syndrome (BBS). Now, BBS patients who are…
Inherited retinal diseases (IRDs) are a group of genetic ocular disorders characterized by vision loss or blindness. A few examples are retinitis pigments (RP) and Usher syndrome. There has been…
From June 11th until the 14th, the Society of Nuclear Medicine & Molecular Imaging (SNMMI) Annual Meeting was held in Vancouver, Canada. At this meeting, Attralus Inc. shared new data…
Andrew is a pretty tough kid - he had already undergone four open heart surgeries by the time he was three years old. Now, at age 21, he's added two…
Recently diagnosed acute myeloid leukemia (AML) patients with IDH1 mutations now have a new treatment option, as the FDA has granted approval to TIBSOVO (ivosidenib tablets)! This is an expansion…
Long-term extension (LTE) data is now available for setmelanotide, which is a treatment for rare causes of obesity, such as Bardet-Biedl syndrome (BBS) and POMC or LEPR deficiency. Data on…
Advocacy is an extremely important part of supporting and helping the rare disease community. A big part of this is rare disease advisory councils (RDACs), which are governmental groups comprised…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
During this month, the Dravet Syndrome Foundation (DSF) organizes a variety of activities and resources to help raise awareness for Dravet syndrome, a rare form of epilepsy. They make it…
If you've been paying attention to the news lately, you know there's a shortage of baby formula in the United States. Many, many mothers and children have been affected, leaving…
Every year, people from across the world come together to celebrate Myasthenia Gravis Awareness Month during the month of June. The Myasthenia Gravis Foundation of America (MGFA) plays a big…
Chinese patients living with pediatric growth hormone deficiency are one step closer to a new treatment option, according to a recent article from PharmaBiz.com. The step I'm referring to is…
Acromegaly is a rare disease that is characterized by excess and abnormal levels of growth hormone (GH). Because of this, medical professionals are very interested in these levels as patients…
Nalla Lawson, who lives in Ocala, Florida, recently shared her story and treatment journey through an article published in UF Health. After suffering a fall back in 2016, she was…
According to a press release that can be found on AP News, new data have been released on a potential treatment - called NVG-111 - for both mantle cell lymphoma…
The U.S. Food and Drug Administration (FDA) has recently granted approval to tisagenlecleucel, which is marketed under the name Kymriah, for adult patients with relapsed or refractory (R/R) follicular lymphoma…
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