According to a story from Charcot-Marie-Tooth News, the drug company DTx Pharma has recently obtained critical funding that will go towards the development of new RNA-based treatments for Charcot-Marie-Tooth disease…
On October 22, 2020, BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from a Phase 3 clinical trial evaluating berotralstat for patients with hereditary angioedema (HAE). As a…
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…
Earlier this week, the FDA granted Priority Review status to use a combination of Opdivo-Cabometyx as a treatment for patients with renal cell carcinoma (RCC). According to Healio, Opdivo (nivolumab)…
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,…
At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…
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…
When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…
According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by…
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.…
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.…
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