In our third installment from our series of reports provided by our partner TREND Community, we are bringing you several reports that analyze the effects of sleep disorders. This week…
Ireland has unveiled a new national strategy aimed at transforming the diagnosis and care of the estimated 300,000 people in the country living with rare diseases. According to The Irish…
Families and researchers in North Carolina are making urgent appeals to the FDA, highlighting the desperate need for faster approval of drugs for rare and ultra-rare diseases that threaten children’s…
Eli Lilly’s breast cancer drug Verzenio (abemaciclib) has achieved a significant milestone, with new study results showing it helps people with a common form of early-stage breast cancer live longer.…
A new RNA-based drug from Regeneron Pharmaceuticals and Alnylam Pharmaceuticals has shown positive results in a late-stage clinical trial for generalized myasthenia gravis (gMG), a rare, chronic autoimmune disease that…
A recent Phase I/II clinical study led by UMass Chan Medical School has shown encouraging results for a gene therapy targeting GM2 gangliosidosis, a group of rare and fatal neurodegenerative…
A groundbreaking study from the Weizmann Institute of Science and Sheba Medical Center has brought renewed hope for children with kaposiform lymphangiomatosis (KLA), a rare and life-threatening genetic disorder of…
South Africa is on the brink of a major shift in HIV prevention, with plans to introduce lenacapavir—a twice-yearly injectable—at select public clinics by April 2026. As reported by All…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
HIV-associated facial lipodystrophy, a frequent complication of long-term antiretroviral therapy (ART), is characterized by abnormal fat distribution in the face and neck. This condition, often presenting as midfacial hollowing and…
The landscape of HIV treatment continues to evolve, and a significant milestone has just been reached with the FDA’s approval of Rukobia (fostemsavir), a novel therapy from ViiV Healthcare. Reported…
A recent report from Yahoo News highlights the emergence of Guillain-Barré Syndrome (GBS), a rare but serious paralytic disorder, as a growing concern within the humanitarian crisis unfolding in Gaza.…
Baylor Genetics is set to showcase groundbreaking innovations in RNA sequencing for rare disease diagnosis at the 2025 Advances in Genome Biology and Technology (AGBT) Precision Health Meeting, according to…
In our second installment from our series of reports provided by our partner TREND Community, we are going to be focusing on a different condition over four weeks. This week…
The U.S. Food and Drug Administration (FDA) is reviewing the first medication specifically developed for Barth syndrome, a rare and life-threatening genetic disorder. As reported by STAT, this medication could…
A recent report from NDTV World brings attention to a rare but significant public health event: an American man has tested positive for plague, the infectious disease historically known for…
Health authorities in Queensland, Australia, have issued a public warning after the discovery of Naegleria fowleri—a rare but deadly “brain-eating” amoeba—in a town’s water supply. As reported by The Medical…
Rocket Pharmaceuticals has received the green light from the US Food and Drug Administration (FDA) to resume its pivotal Phase II Danon disease gene therapy trial, according to a recent…
Patient Worthy is excited to share our most recent episode of our podcast Wait, How Do You Spell That? featuring Lisa Batista. Lisa shares her story with us of living with…
ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…
A recent study highlighted in Hematology Advisor explores the challenges of monitoring patients with hemophilia A after gene therapy, specifically focusing on the reliability of laboratory assays used to measure…
The Rady Children’s Institute for Genomic Medicine (RCIGM) has announced a landmark international collaboration with Sidra Medicine in Qatar to launch the BeginNGS® newborn genome sequencing program—marking a significant step…
According to a recent article from JournalFeed, the relationship between type 2 diabetes and metabolic dysfunction-associated steatotic liver disease (MASLD) is increasingly coming to the forefront of clinical care. MASLD,…
A routine evening became pivotal for a Portsmouth family when a bright white reflection appeared in nine-month-old Nora’s left eye under bathroom lights. As was reported by Mirror, what seemed…
The Alpha-1 Foundation (A1F) has announced a groundbreaking advancement in the fight against Alpha-1 Antitrypsin Deficiency (AATD) with the launch of AlphaDetect, a dedicated non-profit subsidiary focused on revolutionizing the…
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