The FDA has recently approved of GlaxoSmithKline's (GSK) treatment for lupus nephritis (LN), BENLYSTA. This therapy is the first of its kind, meant to treat systemic lupus and adults with…
Albireo has recently initiated their Phase 3 ASSERT trial, according to GlobeNewswire. This study will evaluate odevixibat as a treatment for Alagille syndrome. This therapy fits well with Albireo's mission,…
Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
According to BioMedWire, biopharmaceutical company AzurRx BioPharma ("AzurRx") decided, in mid-November, to advance a series of clinical trials evaluating MS1819 for patients with cystic fibrosis. In particular, the clinical…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that occurs due to loss of motor neurons, which are the nerve cells that…
by Lauren Taylor from In The Cloud Copy Sickle cell disease or SCD is a type of red blood cell disorder in which the normally round red blood cells are…
by Lauren Taylor from In The Cloud Copy Nephrologists worldwide have shown that there is a huge unmet need when it comes to the treatment of various glomerular diseases, in…
Alina Citovic was diagnosed with spinal muscular atrophy at age five, just three years ago. At the time of her diagnosis, Spinraza was already approved by the FDA. While the…
by Lauren Taylor from In The Cloud Copy Spondyloarthropathies is an umbrella term for a group of chronic diseases that affect the joints. These can affect children and adults and…
The Reuters Pharma & Patient USA 2020 virtual conference included a number of shorter presentations that focused on specific approaches to help drug companies better meet the needs of patients.…
Kura Oncology, a San Diego biopharmaceutical company, was recently featured in a Globe Newswire article, announcing its first-in-human dose escalation of KO-539 being evaluated in the Komet-001 trial (NCT04067336). The…
According to a story from SMA News Today, a recent case study has reported instances in two spinal muscular atrophy (SMA) patients in which repeated administrations of the therapy nusinersen…
According to a story from GlobeNewswire, the biopharmaceutical company MediciNova, Inc. has announced encouraging results related in Optical Coherence Tomography (OCT) in a phase 2b clinical trial. This clinical trial…
In early December 2020, the American Society of Hematology (ASH) held their Annual Meeting and Exposition. During the meeting, many companies presented data on clinical trials and treatments related…
.jpg)
Since March, most of the globe has been anxiously awaiting updates on COVID-19, the virus that has infected over 74 million people. One update was just released: the CDC has…
According to an NBC AP news item, early results show that CRISPR, a “tool” that alters DNA permanently in blood cells, has the potential to not only stop the progression…
According to a story from Markets Insider, the biotechnology company REGENXBIO Inc. has recently released data from its phase 1/2 clinical trial. This is interim data from cohorts 1 and…
According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US…
Xencor, a biopharmaceutical company that focuses on curating treatments for cancers and autoimmune conditions, has recently released data on vibecotamab. This acute myeloid leukemia treatment was included in a Phase…
According to a story from Cancer Therapy Advisor, a recent phase 1 clinical trial investigated an experimental CAR T-cell therapy in heavily pretreated patients living with multiple myeloma, a rare…
As of last month, a newborn screening advisory committee from the Virginia Department of Health declined to add Krabbe disease to the conditions that doctors look for when a child…
According to a story from Newswise, the early results of a recent phase 1/2 clinical trial have revealed the potential of an experimental treatment that was being tested for blastic…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a disease that affects the patient’s central nervous system, peripheral nervous system, and skeletal muscles or…
The New England Journal of Medicine recently carried a report citing data from a Phase 3 clinical trial involving 307 people diagnosed with MSI-H-dMMR metastatic colorectal cancer. MSI-H (high levels…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
