After years of determined advocacy and scientific perseverance, Stealth BioTherapeutics has achieved a landmark victory with the FDA’s accelerated approval of elamipretide (now branded as Forzinity) the first-ever treatment for…
Gomekli (mirdametinib) has become the first FDA-approved treatment for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN)—tumors that often cannot be surgically removed. While…
Avacopan, a novel oral therapy for antineutrophil cytoplasmic antibody-associated vasculitis (ANCA-AAV), has changed treatment paradigms for this rare but serious disease. However, until now, a validated technique for measuring avacopan…
Neurofibromatosis Type 1 (NF1) is a genetic disorder diagnosed primarily through a set of clinical criteria established by the National Institutes of Health (NIH). Typically, a clinical diagnosis of NF1…
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…
In the heart of south Wales, a small but growing community is grappling with the devastating impact of Dentatorubral-pallidoluysian atrophy (DRPLA), a rare inherited neurological disorder that, until recently, was…
Assertio Holdings has announced the results of the first real-world evidence study evaluating patient experience with SYMPAZAN® (clobazam) oral film, presented at the 150th Annual Meeting of the American Neurological…
aTyr Pharma suffered a major setback on September 15, 2025, as its stock price plummeted more than 80% following disappointing results from a pivotal Phase 3 trial of its lead…
In a breakthrough study, scientists have uncovered a surprising ally in the fight against cancer: the bacteria living within tumors. According to World Pharma News, an international team led by…
A new study conducted at the Huntsman Cancer Institute in Utah suggests that exercise has a beneficial effect on the body, such as reprogramming, that alters the gene’s function, identity…
A newly developed assessment method that brings together a blood-based measurement and a physical body metric is showing promise for forecasting both survival chances and nutritional health in patients battling…
Dewpoint Therapeutics, a U.S.-based biotechnology innovator, has announced the successful closure of its Series D financing round. As reported by thepharmaletter, Dewpoint Therapeutics is renowned for its pioneering work in…
Palvella Therapeutics, Inc. has announced a key milestone in the development of therapies for rare genetic skin diseases: the completion of enrollment in its Phase 2 TOIVA trial of QTORIN™…
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…
Health Canada’s recent approval of adjuvant (applied after surgery) Keytruda (pembrolizumab) in high-risk invasive cancer of the bladder saw patients remain free of cancer in double the current standard, according…
Capsida Biotherapeutics has suspended a clinical trial of its experimental gene therapy after the first child to receive the treatment died, sending shockwaves through the rare disease and gene therapy…
Glioblastoma is notorious as the most lethal form of malignant brain tumor, with most patients surviving just one or two years after diagnosis. Part of what makes these tumors so…
A recent milestone in gene therapy is transforming the outlook for people with rare genetic skin disorders, signaling a new era of personalized medicine and hope for patients who previously…
Research into rare diseases has long faced obstacles, from limited patient numbers to the challenge of replicating disease biology in the lab. A new wave of innovation, highlighted in the…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
The U.S. Food and Drug Administration (FDA) has granted an expanded indication for Vonvendi, Takeda’s recombinant von Willebrand factor (rVWF) therapy, marking a significant advancement in the management of rare…
Stanford University has recently secured a prestigious grant from the U.S. National Institutes of Health (NIH) to accelerate the clinical development of Tiakis Biotech’s novel therapy, Elafin (also called Tiprelestat),…
Pfizer has announced breakthrough Phase 3 results for HYMPAVZI™ (marstacimab), a once-weekly subcutaneous therapy that could redefine care for people with hemophilia A or B who have developed inhibitors, as…
Gene therapy for hemophilia B has reached a significant milestone: more than a decade after a single treatment, patients continue to experience stable benefits with minimal safety concerns, according to…
For families affected by rare genetic skin diseases, hope once meant daily bandages and vigilant wound care. That began to change in 2023, when US regulators approved the first-ever gene…
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