Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
According to a story from Bioportfolio, trials testing the treatment inclisiran are reaching their enrollment goals ahead of schedule. Inclisiran is currently being tested to reduce LDL-cholesterol in a variety…
Doctors of semi-professional soccer player, Matt Dimbylow, believes he developed Parkinson's disease at the young age of 36-years-old due to a head injury he sustained playing the sport, reports Mirror.…
Researchers from Duke Health are hoping to discover new treatments for Lyme disease since the only way to currently manage the disease is by taking consistent antibiotics, which can be…
According to an article from GlobeNewswire, new clinical evidence supports the use of gelpaglutide as a once-weekly treatment for short bowel syndrome. Zealand Pharma completed a Phase I clinical trial…
Rett syndrome might limit 8-year-old Laurel Cooper's ability to speak and do every day tasks, but it doesn't hold back her spunky, sassy attitude, reports University of Wisconsin-Madison News. She will…
Originally reported by the Vancouver Sun, children from Langley B.C. are standing up to request access to a potentially life-changing drug as they face the day to day pain onset…
According to an article from FinancialBuzz, the pharmaceutical company Avadel announced that its product FT 218 has been granted orphan drug status by the Food and Drug Administration (FDA) as…
Researchers from St. Jude Children's Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID,…
Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…
Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…
A recent study conducted by researchers from National University of Singapore Medicine laboratory shows that eating vegetables, specifically broccoli, might prevent and even kill cancer cells that develop into colorectal…
A little more hope is given to individuals suffering from Lennox-Gastaut syndrome and Dravet syndrome as GW Pharmaceuticals shared the U.S. FDA will indeed be accepting their application for potential…
Many cancer and blood specialists will not have encountered systemic mastocytosis. A new drug targets the underlying defect of the disease and will help put doctors on the lookout. Keep…
Most disabilities in Pakistan are caused by three diseases: Spinal Muscular Atrophy, Spinal Cord Injury, and stroke. With this in mind, a group in the country’s capital has launched the…
David Fogel's aunt was diagnosed with an advanced stage of stomach cancer when she was just fifty-six years old. Constantly dealing with severe pain and the side effects of treatment,…
Out-of-pocket expenses for rare disease are astronomical, and the Ministry of Health (MOH), is taking time to find a more positive way to help benefit children and their families burdened…
The FDA approved a human study to be conducted by Swedish Orphan Biovitrum AB to treating mucopolysaccharidosis type lllA patients with a new drug called SOB1003, reported European Pharmaceutical Review. Not only…
Scientists in the UK are hopeful that they have discovered a new tool in the battle against brain cancer. The surprising solution is a virus. Ten patients in the UK…
According to a story from IBD News Today, the Food and Drug Administration (FDA) has just issued approval for the pharmaceutical company Immunic Therapeutics to move forward with Phase II…
According to a story from PR Newswire, the pharmaceutical company Biohaven announced that they have received clearance from the Food and Drug Administration (FDA) to begin work on its clinical trials…
Patients suffering from the rare blood disease, hemophilia, deal with daily demands of their treatment. A recent study supports self-infusion for these patients, reported Harrison Daily Times. Self-infusion is when…
On January 17th, Parent Project Muscular Dystrophy announced that the first patient had been treated with a new gene therapy. The therapy developed specifically for Duchenne muscular dystrophy is known as…
According to an article from rdmag.com, a new form of treatment could allow for organ tissue to regrow to a normal level in patients with conditions in which organs are…
The gene therapy development company, AveXis, Incorporation, just announced that they will be expanding their clinical trial development program for the potential gene therapy treatment: AVXS-101, reported Globe News Wire.…
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