Clinical Trials
Recent Posts
- FDA OKs Trial of Investigational DM1 Therapy
- Advocates Lobby US Congress During Virtual Rare Disease Week
- The People We’ve Met Due to My Son’s Disability Are a Blessing
- US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing
- Free DMD Newborn Screening Now Offered at Boston Hospital
- Rare Myopathy Affects Women, Too, Advocate Emphasizes
- Blood Pressure Medicine Combo Helps Slow Heart Decline in DMD Study
- From ‘Motor Moron’ to a Limb-Girdle MD Diagnosis
- Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds
- DMD Trial Testing Oral ASP0367 Recruiting Boys 8–16 in US