In early October, the FDA approved an Investigational New Drug Application (IND) for APR-548, a potential treatment for patients with TP53 mutant myelodysplastic syndromes (MDS). According to a press release from…
According to a story from BioSpace, SIRION Biotech GmbH and Mustang Bio, Inc. have announced a new agreement for licensing the use of SIRION's proprietary LentiBOOST™ technology for use by…
According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will…
According to a story from Newswise, it was more than three years ago that Maxford Brown, 16, awoke one morning feeling out of sorts. Maxford, who lives with Down syndrome,…
In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While…
Many people within the rare disease community share a common experience: feeling isolated or misunderstood because of your condition. In some cases, such as conditions causing chronic pain or fatigue,…
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…
Ovid Therapeutics has recently announced that it will host a webinar in an effort to educate people about Angelman syndrome and their possible treatment for it, OV101. Speakers will focus…
According to a story from BioSpace, the biopharmaceutical company Kite announced recently that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has released…
According to a recent article in Healio, Therapeutic Options for Interstitial Lung Disease was one of the topics covered at the Congress of Clinical Rheumatology 2020. Dr. Elizabeth Volkmann co-director…
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…
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