Priority Review Granted for Belzutifan NDA for VHL-Associated RCC
For nearly 130 years, pharmaceutical company Merck & Co ("Merck" or "MSD") has been working to develop unique therapeutic solutions for those with rare cancers and a variety of…
Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
For nearly 130 years, pharmaceutical company Merck & Co ("Merck" or "MSD") has been working to develop unique therapeutic solutions for those with rare cancers and a variety of…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
In early March 2021, the National Comprehensive Cancer Network (NCCN) shared a new set of NCCN Guidelines for Histiocytosis. Histiocytosis, or hystiocytic disorders, are a group of rare conditions characterized…
In mid-March 2021, Mitsubishi Tanabe Pharma Canada ("MTP-CA") shared that its infusion therapy RADICAVA (edaravone) is now available for some Canadian patients with amyotrophic lateral sclerosis (ALS). The treatment will…
Over the last year, news of COVID-19 has dominated the headlines. Caused by SARS-CoV-2, a coronavirus, the global pandemic is now associated with 116 million diagnoses worldwide and 2.57 million…
First formed and launched within the UPstart incubator at the University of Pennsylvania's Penn Center for Innovation (PCI), biopharmaceutical company Linnaeus Therapeutics, Inc. ("Linnaeus") is now making strides in the…
Is it possible for certain gene mutations, which cause a specific condition, to also raise the risk of developing another condition? Well, says HealthDay, the answer might be yes. In a…
At first, everything in Amberlyn Smith's pregnancy seemed to be going smoothly. When she was just over 29 weeks pregnant, her screening tests appeared to be normal. However, at…
Patients with hereditary angioedema (HAE) experience recurrent "attacks," or periods of swelling under the skin. Now, in a Phase 2 clinical trial, researchers are evaluating PHVS416 for patients with…
In a recent press release, biopharmaceutical company Hongkong Winhealth Pharma Group Co., Ltd. ("Winhealth") announced a new strategic cooperation agreement between itself and Merz Pharmaceuticals GmbH ("Merz"). Altogether, this agreement…
Past research has shown up to 83 genes and genetic loci associated with glaucoma. However, reports the Massachusetts Eye and Ear Infirmary in a news release, new research has identified…
On March 11, 2021, global biopharmaceutical company Alkermes plc shared that its investigational drug candidate nemvaleukin alfa received Orphan Drug designation from the FDA. The treatment is designed for patients…
Altogether, there are over 7,000 rare diseases. Worldwide, 1 in 20 people live with a rare disease. But the journey to diagnosis can sometimes be confusing or difficult. So when…
In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease…
Biopharmaceutical company OncoSynergy recently shared that the first patient was dosed in a Phase 1 clinical trial. Within this trial, researchers are exploring OS2966 for patients with recurrent glioblastoma.…
In a recent press release, the Ivy Brain Tumor Center, a nonprofit translational science initiative, shared that the first patient was treated in a Phase 0 clinical trial. The clinical…
According to Healio, giant-cell arteritis (GCA) is seeing a worrying trend. Over a period of 18 years (2000-2018), mortality rates for GCA increased from 50 to 57.6 per 1,000 deaths.…
At the end of February 2021, BridgeBio Pharma Inc. ("BridgeBio") and its affiliate Origin Biosciences, Inc. ("Origin") shared that its treatment, NULIBRY (fosdenopterin) for Injection, received FDA approval for patients…
If you've ever looked into the field of medical research, you may have heard the term biomarker. In short, a biomarker is a type of measurable sign or indicator which suggests…
Each year, over 200 million people worldwide, many in Africa and South Asia, are diagnosed with malaria. Unfortunately, this mosquito-borne illness is also fatal for thousands. While some countries are…
In 2015, the FDA approved Apollo Endosurgery, Inc.'s Orbera Intragastric Balloon for weight loss. Now, the company shares in a press release, the device recently received Breakthrough Device Designation…
For more than 30 years, Ionis Pharmaceuticals, Inc. ("Ionis") has worked to create RNA-targeted therapies for patients in need. Now, according to a recent press release, the company initiated…
Some cancers and hematological conditions can be difficult to treat. But biotechnology company Oncopeptides AB is working to address this issue for patients with relapsed or refractory (R/R) multiple…
The FDA's Orphan Drug designation offers a special status to drugs or biologics designed to treat rare conditions. In the United States, a rare disease is one that impacts less…
In a recent press release, biopharmaceutical company Salarius Pharmaceuticals, Inc. ("Salarius") shared that it had initiated an expansion stage of a Phase 1/2 clinical trial. Within the trial and the…