Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
With each new day and technological improvements come new discoveries about the world around us. Some of those discoveries center around medical conditions which have never been seen before. According…
According to the Daily Sabah, the Health Ministry of Turkey is taking a new approach to family planning and screening for genetic disorders. As of October 8, 2021, the Health…
Dimeric calpains are calcium-activated cysteine proteases which help to regulate cell function. If these calpains are overactive, it could lead to fibrosis (tissue scarring) and tissue damage. In the…
Continue ReadingNew Optimism for Treating Machado-Joseph Disease
Most patients with Norrie disease, a rare X-linked genetic disorder, often develop sensorineural hearing loss. But what mechanisms cause this hearing loss to occur? According to Medical XPress, researchers from…
Continue ReadingNDP Deficiency Causes Deafness in Norrie Disease
At just 18 weeks old, Ivy Gaines, who was born prematurely, was diagnosed with spina bifida. His parents, Robert and Brittney, have worked tirelessly to ensure that Ivy is receiving…
Continue Reading“Ride 4 Ivy” Raised Funds for Son with Spina Bifida
The Child Growth Foundation (CGF) is a leading UK charity which supports families, adults, and children with rare growth conditions such as SHOX deficiency, Silver Russell Syndrome, and Sotos syndrome.…
Continue ReadingFather Runs to Fundraise for Sotos Syndrome Awareness
Fenchol, a natural compound, is known for its herbal and appealing scent. This compound is found in a number of plants, including basil, nutmeg, and eucalyptus leaves. In the past,…
Continue ReadingFenchol, Found in Basil, Could Protect Against Alzheimer’s
To best address the needs of patients with rare diseases and rare cancers, additional treatment options are necessary. But developing these treatment options can be time-consuming and sometimes difficult.…
Continue ReadingIMX-110 for Soft Tissue Sarcoma Earns Orphan Drug Designation
Screening tests are used to identify potential signs related to certain diseases or conditions. For example, newborn screening, performed at birth, gives insight into whether or not a child has…
Continue ReadingFirst Colorectal Cancer Screening Now Recommended at Age 45
Ticks can carry a variety of infectious pathogens. Once the ticks bite humans, humans can then be infected with illnesses ranging from babesiosis and Powassan disease to Lyme disease. Now,…
If you could receive a vaccine to potentially prevent any condition, or at least reduce its severity, would you? According to Medical XPress, University of Toledo researchers are exploring this…
Continue ReadingExperimental Vaccine Could Prevent Rheumatoid Arthritis
Recently, biopharmaceutical company PTC Therapeutics, Inc. performed a five-year data analysis to evaluate the continued safety, efficacy, and tolerability of PTC-AADC. The company developed this novel gene therapy to improve…
Continue ReadingPTC-AADC Shows Benefits for Patients with AADC Deficiency, Data Shows
According to Healio, the FDA recently granted Orphan Drug designation to nadunolimab (CAN04), a treatment candidate for patients with pancreatic cancer. Orphan Drug designation is given to drugs or biologics…
Continue ReadingNadunolimab Scores Orphan Drug Designation for Pancreatic Cancer
The current standard-of-care for children with cerebral palsy often consists of a blend of occupational and physical therapy. But what if another therapy could be more beneficial? Medical XPress describes…
Continue ReadingCIMT Improves Mobility, Skills for Children with Cerebral Palsy
If you've ever heard any discussions around the topic of opioids, you probably know that there is a lot of controversy and a similar deal of complexity. However, researchers in…
Continue ReadingStudy Explores the Effects of Opioids on Inflammatory Bowel Disease
What would you do to support your child? Would you take on a daunting 100 kilometer (62 mile) run? According to the Daily Record, a mother and her friends in…
Continue ReadingMom and Friends Run for Sturge-Weber Syndrome Awareness
In the past, there have not been any available treatments to tackle cholestatic pruritus, or a severe and unrelenting itch, for patients with liver diseases such as Alagille syndrome. However,…
Continue ReadingLIVMARLI Becomes First FDA-Approved Drug for Alagille Syndrome Pruritus
Clinical trials are important to understanding both more about certain conditions and more about the safety, efficacy, and tolerability of drugs designed to treat these conditions. Currently, biopharmaceutical company AMO…
Continue ReadingFirst Patient Joins AMO-02 Trial for Congenital DM1
The FDA created its Orphan Drug Act to incentivize drug developers to develop solutions for patients with rare or life-threatening conditions. Now, Orphan Drug designation is granted to drugs or…
Continue ReadingMarzAA Earns Orphan Drug Designation for Factor VII Deficiency
In the past, caffeine has been somewhat of a complex or even controversial topic. While some research shares that it is bad for health, others laud caffeine’s ability to enhance…
Continue ReadingCaffeine Intake Not Linked to Invasive Breast Cancer, Study Shares
In the United States, Orphan Drug designation is granted to drugs or biologics which show promise in treating patients with rare and life-threatening conditions. For the purpose of this article…
Dave and Kendra Riley have a beautiful family: them and their daughters Eva, Kiera, and Olivia. Yet the family has faced some scary and difficult obstacles over the last…
Continue ReadingAhwatukee Family Raises Awareness of Metachromatic Leukodystrophy
Clinical trials have many endpoints - or milestones they are looking to achieve. These endpoints help signify the safety, efficacy, and tolerability of the treatments. In the Phase 3…
Continue ReadingKEYTRUDA Met OS Endpoint in Hepatocellular Carcinoma Clinical Trial
Sometimes, certain drugs may be approved in different countries and not in others. However, drug approval spanning multiple countries highlights the importance of getting drugs and treatments into the hands…
Continue ReadingTAVNEOS Approved in Japan for ANCA-Associated Vasculitis
I don’t know about you, but I always get incredibly happy when I see stories about a community rallying to support one of its own members. For example, those in…
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