Growing up, I guess you could say I was living my childhood dream. I was very close with my mother, was well liked by my peers, and did pretty decent…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
by Lauren Taylor from In The Cloud Copy Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease of the blood in which the affected individuals red blood cells break apart.…
The road to diagnosis for a rare disease patient is often a long one. Countless doctors, appointments, and tests are typically needed to finally discover the cause behind one's symptoms.…
A study recently published in Cell Stem Cell has shown that using human stem cells in combination with medical chemistry can improve existing medications to treat genetic disorders. Dr. Alfred George Jr.…
by Lauren Taylor from In The Cloud Copy A comorbidity occurs when there is a presence of one or more additional illnesses or conditions that co-occur with the primary condition.…
by Lauren Taylor from In The Cloud Copy Latent autoimmune diabetes in adults (LADA) is a less often talked about form of diabetes that occurs as the result of the…
According to a story from BioSpace, a recent study conducted by the biotechnology company Goldfinch Bio in collaboration with academic partners is highlighting the importance of proteinuria in determining outcomes…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
Takeda Pharmaceuticals has recently announced the results from their open-label extension study, titled VISIBLE, which evaluates Entyvio as a treatment for ulcerative colitis. According to BioSpace, these results maintain the…
by Lauren Taylor from In The Cloud Copy A local Florida teenager has inspired her whole city after a sudden diagnosis with a serious neurological condition: Chiari malformation. Jordan Ray…
According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…
As reported in Biospace, the National Organization for Rare Disorders (NORD) has just opened registration for patients to take part in a natural history study on metachromatic leukodystrophy (MLD), a…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to Healio, medical professionals from Boston Children's Hospital have conducted research that provides a better look at systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease. They looked specifically…
According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…
Amolyt Pharma has recently announced that they have dosed the first patient in their trial of AZP-3601, a treatment for hypoparathyroidism. According to GlobeNewswire, this treatment will adequately address the…
According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…
Fetal alcohol spectrum disorder (FASD) is a spectrum of disorders that can impact decision making and make individuals unable to understand the consequences of their actions. Because of this, it…
EB Research Partnership (EBRP) is based out of New York, United States. EB Research Foundation (EBRF) is an organization based out of Australia with the same goals. The two organizations have…
On October 14th, 2020, the International Waldenström's Macroglobulinemia Foundation (IWMF) hosted a webinar featuring Dr. Irene Ghobrial of the Dana Farber Cancer Institute. The focus of her presentation was on…
In December 2019, the FDA approved Ervebo, the first-ever Ebola vaccine. However, despite preventative measures, there still was never any actually approved treatment options - until now. Recently, the FDA…
Over this past week, the Harrington Discovery Institute announced five recipients of its introductory Harrington UK Rare Disease Scholar Award competition. The Institute, which is involved with Cleveland's University Hospitals,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
About one month ago, the HealthWell Foundation announced the beginning of the Adrenal Insufficiency Fund. Designed to assist patients with adrenal insufficiency, the fund will grant financial assistance to…
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