AdvocacyRSSToday
A personal story about a man named Jared who has severe hemophilia B (a bleeding disorder) and seizures since childhood. The article highlights how teachers and classmates treated him as 'special' or different on his first day of fifth grade, showing how invisible illnesses can affect children socially and emotionally, not just physically.
WHY IT MATTERSChildren with hemophilia B and other invisible illnesses often face social stigma and misunderstanding in school settings, which can impact their mental health and quality of life as much as the medical condition itself.
Clinical trialUNITERAREToday
Novartis is looking for adults who have immune thrombocytopenia (ITP) or warm-antibody autoimmune hemolytic anemia (wAIHA) — two blood disorders where the immune system attacks blood cells — and who previously did well on a drug called ianalumab. This new study will test whether ianalumab continues to work for these patients. The trial is expected to start in April 2026.
WHY IT MATTERSThis trial is recruiting adults who previously responded to ianalumab for ITP or wAIHA, offering a potential path to continued access for patients whose symptoms improved on this treatment.
AdvocacyRSS5 days ago
This article is a personal account from someone living with both Parkinson's disease and bipolar disorder at the same time. The author discusses how having these two conditions together creates unique challenges for their mental and physical health. The piece highlights the importance of addressing mental health alongside neurological symptoms in Parkinson's care.
WHY IT MATTERSPatients with Parkinson's disease who also have bipolar disorder often face overlooked mental health needs, as treatment plans may focus primarily on movement symptoms while psychiatric care gets deprioritized.
Clinical trialUNITERAREApr 5
Researchers are testing a new drug called pacritinib in people ages 12 and older who have myelodysplastic syndromes or myelodysplastic/myeloproliferative neoplasms — rare blood disorders where the bone marrow doesn't make enough healthy blood cells. The drug works by blocking several proteins that may be causing these diseases. This is an early-stage study sponsored by the National Cancer Institute that will start recruiting patients in April 2026.
WHY IT MATTERSThis trial offers patients with myelodysplastic syndromes access to a novel multi-targeted kinase inhibitor that may address disease mechanisms not covered by current standard treatments, with enrollment beginning in April 2026.
ResearchCLINICALTRIALSMar 30
This study is looking for 2,800 people to help researchers understand how being a caregiver affects a person's health and stress levels over time. The study includes caregivers of people with rare diseases like Batten disease, Tay-Sachs disease, and other inherited metabolic disorders, as well as caregivers of people with undiagnosed diseases. By learning more about caregiver stress, researchers hope to find ways to better support the millions of unpaid family caregivers in the U.S.
WHY IT MATTERSIf you or a family member care for someone with a rare inherited metabolic disorder, Batten disease, Tay-Sachs, or an undiagnosed disease, this study directly addresses the health challenges caregivers face and could lead to better support resources.