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.…
According to a story from Vice World News, the Solanki family, who are residents of Ahmedabad, the largest city in India's western state of Gujarat, recently took action to save…
In a recent press release the US Food and Drug Administration (FDA) has officially approved remdesivir (marketed as Veklury) as a treatment for cases of COVID-19 that require hospitalization. It…
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.…
According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug…
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.…
Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the…
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…
On October 15th, 2020, the Rare Disease Legislative Advocates (RDLA) held a webinar discussing some of the latest news regarding ongoing legislation that is most relevant to the rare disease…
by Lauren Taylor from In The Cloud Copy The human papillomavirus (HPV) vaccine has been being administered to teens and young adults worldwide for over a decade now. It has…
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…
What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…
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…
by Lauren Taylor from In The Cloud Copy Polycystic ovarian syndrome or PCOS is a disorder in which women of reproductive age experience irregular menstrual cycles and/or excess androgen (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…
by Lauren Taylor from In The Cloud Copy Cervical dystonia, sometimes called spasmodic torticollis, is a condition in which the affected individual’s neck muscles involuntarily contract which causes the head…
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…
/ Duke Researchers Create a Type 1, Type 2 Lupus Disease Model Duke Researchers Create a Type 1, Type 2 Lupus Disease Model October 20, 2020 Print-Friendly Version / Save…
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).…
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