A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
According to CureDuchenne, two companies have began a collaboration in an effort to create new gene therapies for Duchenne muscular dystrophy (DMD). Ultragenyx and Solid Bioscience are both using their…
Early on October 28, 2020, Insmed Inc. ("Insmed") announced that it received Marketing Authorization from the European Commission for ARIKAYCE. This therapy is designed for adult patients with nontuberculous mycobacterial…
Colton’s Story Debbie Moore was 18 months old when she was diagnosed with X-linked hypophosphatemia (XLH) a rare, deforming, and painful bone disorder. XLH causes softening of the bones…
Novartis has recently released interim data on iptacopan, according to a press release. The data on the C3 glomerulopathy treatment was released at the American Society of Nephrology 2020 Annual…
In a recent study published in Clinical and Experimental Immunology, researchers determined that patients with high ANCA antibody levels are at an increased risk of having ANCA-associated vasculitis (AAV) relapses. According…
Patient-Focused Drug Development Meeting with the FDA Organized by the National Organization for Rare Disorders (NORD), this meeting will be a critical opportunity for patients to communicate directly with regulators…
Researchers at the Mayo Clinic have collaborated with the international community to develop a potential way to test for spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph Disease. Additionally, they…
A recent study published in PLOS ONE has reinforced the importance of vaccinations specifically for those already fighting a rare disease. This study was written by Sylvie Lesage from the…
Many different approaches have been offered by scientists to stem the overwhelming tide of Alzheimer’s disease (AD). Fifty percent of primary care physicians believe that the medical profession is not…
Monitoring tumors is an essential part of cancer treatment; it allows doctors to make informed decisions regarding which therapies to use. For many forms of cancer, medical professionals have been…
A new treatment for Duchenne muscular dystrophy (DMD) has recently been approved by the FDA. The therapy is called viltolarsen and it was created by Nippon-Shinyaku and the National Center…
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 Myasthenia Gravis News, researchers recently determined a new therapeutic target for myasthenia gravis (MG): activating a bile acid receptor called TGR5. By activating this bile acid receptor, researchers…
Telehealth is something that has become common during the coronavirus pandemic. It allows people to see their physicians without stepping out of the home, drastically lowering the risk of exposure.…
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…
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