Clinical News
Bacteria in Patients’ Guts Show Changes That May Weigh on Disease
People with Parkinson’s disease have substantial changes in the bacteria living in their gut relative to people without this neurodegenerative disorder, an analysis underscores. “This dysbiosis [microbial imbalance] might result in a pro-inflammatory status which...
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Brain Involvement in SMA Type 1 Still Poorly Understood, Review Finds
Children with spinal muscular atrophy (SMA) type 1 may have neurodegeneration in several areas of the brain, with impaired cognitive function and profound speech difficulties, according to a review study. However, brain involvement in this severe type of SMA is...
More Barriers, Lower Socioeconomic Status Tied to Less Exercise Therapy
People with pulmonary hypertension are less likely to be referred to exercise therapy if they are of lower socioeconomic status or perceive more barriers to accessing such therapy, a study shows. These findings highlight the need for increased education about the...
New SMN-boosting Molecule Shows Promise as Add-on Therapy
Targeting a specific region of an intermediate molecule generated from the SMN2 gene increased the levels of functional SMN — the protein lacking in spinal muscular atrophy (SMA) — in cells derived from SMA patients, a study shows. In addition, combining this approach...
Virtual Reality Could Play a Role in the Future of Our Health
This week, I went to Tokyo, saw elephants up close in Africa, flew over New York, and watched a stage show, all in one day. How was it possible? Virtual reality (VR), of course! Being unable to play badminton in lockdown has forced my husband to seek competitiveness...
FDA Clears Potential IPF Therapy, IM156, for Phase 1 Trial
The U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for ImmunoMet Therapeutics‘ treatment candidate for idiopathic pulmonary fibrosis (IPF), IM156. The decision grants ImmunoMet permission to open studies of the...
TOPAZ Trial Results on Muscle Therapy Likely by June, Scholar Rock Says
Efficacy and safety results from the TOPAZ Phase 2 trial evaluating apitegromab, a muscle-directed therapy for spinal muscular atrophy (SMA) being developed by Scholar Rock, are expected by June, the company announced. “2020 was a transformative year for Scholar Rock...
At-home Activity Trackers and Spirometry Devices May Aid IPF Trials, Small Study Finds
At-home use of wearable physical activity trackers and portable spirometry devices may offer a feasible way of evaluating people with idiopathic pulmonary fibrosis (IPF) taking part in clinical trials, a small study suggests. These findings were reported in “Mobile...
More Effort Needed to Recruit Minorities to Phase 3 Clinical Trials, MS Expert Says
Because multiple sclerosis (MS) presentation and progression course can be very different between people of African ancestry and Caucasians, the recruitment of minorities to Phase 3 clinical trials is of particular importance. Researchers in the MS field and the...
We Need to Widen Inclusion Criteria for Cystic Fibrosis Clinical Trials
As readers of my column, you know that I write about the journey of life and the emotions of living with cystic fibrosis (CF). This week, I want to address a topic that makes me sad and frustrated. I’ve made it my mission to shine a light on the current clinical trial...