Ollin Biosciences has announced important clinical trial results showing that OLN324, an investigational eye treatment, outperforms an existing therapy in treating two serious retinal diseases that threaten vision in millions…
As reported on Business Wire, Alkermes plc has initiated a global phase 3 clinical development program to evaluate alixorexton, an investigational oral therapy for people living with narcolepsy type 1…
Editor's Note: This is the second part in a series of excerpts written by Dana Langston. Chapter 4: The Internal Shift We are living in the "waiting room" now. The…
Multiple myeloma, a cancer of plasma cells in the bone marrow, has become an increasingly manageable disease over the past two decades, thanks to successive waves of innovative therapies. However,…
As reported on FierceBioTech, Gilead Sciences has closed one of its midstage clinical studies evaluating an experimental HIV regimen, following ongoing safety concerns that prompted U.S. regulators to intervene last…
Editor's Note: The following is the first part of excerpt written and submitted to us by Dana Langston. There is a specific kind of internal battle that happens when you've…
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
This is Part 3 of three articles I wrote about pain. The first 2 are entitled: Part 1- The real impact of pain that people cannot see Part 2- Life…
My journey with cardiac amyloidosis started shortly after I retired as a firefighter/paramedic in spring 2018. I started with trigger fingers—one in 2018 and two more in 2020. Also in…
While surgery has long been the standard for removing solid tumors, a revolutionary technique called histotripsy is offering an alternative, as reported on MedPage Today. Histotripsy uses high-intensity, focused ultrasound…
Remix Therapeutics has achieved a significant regulatory milestone with the U.S. Food and Drug Administration granting Fast Track designation to REM-422, a first-in-class small molecule therapy designed to treat recurrent,…
My whole life growing up, there was this specific buzzing sound that would come alive on the sunniest of days. I’ve been told over the years it’s because of everything…
On 12/12/2025, I was admitted into the ER for a thrombectomy due to a bilateral pulmonary embolism. While I am overweight and sometimes eat like a raccoon in the trash,…
The American Nutrition Association (ANA) has announced a collaborative initiative with Jazz Pharmaceuticals designed to advance evidence-based nutritional education for individuals living with narcolepsy and idiopathic hypersomnia (IH), sleep disorders…
This patient story is sponsored by Chiesi Global Rare Diseases and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Patients living with chronic or rare diseases and the caregivers coordinating appointments, medications, and daily life often carry a quiet question alongside the medical work: what will last beyond the…
The Dravet Syndrome Foundation of Spain hosted its 2026 Annual Family Meeting on March 20 at the Rafaela Ybarra School in Madrid, bringing together roughly 100 family members, researchers, and specialists.…
The pharmaceutical industry celebrates a significant victory for rare disease patients as the Food and Drug Administration approved Denali Therapeutics' groundbreaking enzyme replacement therapy for Hunter syndrome, marking a pivotal…
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