The FDA has recently provided 89bio with guidance for their upcoming clinical trial of BIO89-100, a treatment for nonalcoholic steatohepatitis (NASH). The trial will advance as planned, beginning in the…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
The European Commission (EC) has granted the Orphan designation to Passage Bio for their Krabbe disease treatment, PBKR03. This adds another designation to the medication's list, as it has already…
According to a story from Markets Insider, the biopharmaceutical company CStone Pharmaceuticals has announced that China's National Medical Products Administration (NMPA) has approved avapritinib (marketed as Ayvakit) as a treatment…
Orphazyme's latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
Spinal muscular atrophy (SMA) patients aged two months and older will now have an at-home treatment option in Europe. The European Commission (EC) has approved of Evrysdi for those who…
By May 25th, 2021 the FDA will announce their decision regarding approval of Opdivo used in combination with chemotherapy for gastrointestinal cancers. Back in December, Priority Review was granted to…
Lyme disease, and other tick-borne illnesses, can cause intense and prolonged symptoms. In fact, some patients experience chronic symptoms for a number of years following the initial bite. However,…
According to a story from express.co.uk, a treatment for phenylketonuria (PKU) is currently under consideration for coverage on the UK's National Health Service (NHS). The treatment is called Kuvan and…
According to a story from businesswire.com, the pharmaceutical company Bayer has recently announced the release of results from its phase IV trial. This trial evaluated the effects of the drug…
Lisa Stockman Mauriello of Summit, New Jersey, is not alone in her struggle to gain access to the company’s experimental ALS drug, tofersen. In fact, according to a recent article…
According to a story from PR Newswire, the biotechnology company Ionis Pharmaceuticals recently announced promising findings from its phase 2 clinical trial. This clinical trial was evaluating the company's experimental…
Rare disease patients face a number of obstacles when it comes to getting the proper diagnosis and treatment. There's a lot of work being done to address these problems, some…
NORD (the National Organization for Rare Disorders), through PR Newswire, recently announced the results of a new study that reveals the total number of generics, orphan products, and biosimilars currently…
Rett syndrome is a rare neurological disorder which often presents within the first 6 months to 1.5 years of a child's life. As the condition progresses, children often lose…
Tanner McLeod has been searching for a stem cell donor for a long time. He was diagnosed with sideroblastic anemia at just six months old and has required a transplant…
Recent results of a Turkish trial have demonstrated the need for further research into IFN-gamma as a treatment for Friedreich's ataxia (FA). This study showed that the therapy resulted in…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
Recently, the Endocrine Society's virtual Annual Meeting, dubbed ENDO 2021, took place from March 20 - 23, 2021. During the meeting, attendees discussed the latest in endocrinology research, treatment,…
At the Endocrine Society's Annual Meeting, or ENDO 2021, Amolyt Pharma ("Amolyt") shared positive clinical data from a Phase 1 clinical trial evaluating AZP-3601 for patients with hypoparathyroidism. Although…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
A recent study published in Arthritis Care & Research illustrated how higher dosages of prednisone lead to better outcomes for lupus nephritis (LN) patients. After one year, participants were found to…
No longer the new player on the team, next-generation sequencing (NGS) is well established as having an influence in clinical care. In recent years, NGS has been responsible for many…
Chronic kidney disease (CKD) affects hundreds of millions of people throughout the world. As of 2017, 697.5 million people were estimated to have CKD by this study published in Nature Reviews…
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