Posts by: Forest Ray PhD

Angioedema News

Use of Takhzyro (lanadelumab) for at least six months led to fewer hereditary angioedema (HAE) attacks and treated attacks, a case review study from Canada reported. Three of its 12 patients ... Read more

September 23, 2021September 23, 2021

New CTSD Gene Variant Not Linked to Rare CLN10 Batten in Case

Batten Disease News

A new mutation found in the CTSD gene does not associate with the rare CLN10 form of Batten disease, despite occurring in a girl in China with CLN10-like symptoms, according to ... Read more

September 17, 2021September 17, 2021

Two Tests May Be Better Than One at Predicting Survival

ALS News Today

The two most common measures used in amyotrophic lateral sclerosis (ALS) assessments — the ALS functional rating scale-revised (ALSFRS-R) and forced vital capacity (FVC) — can predict survival more accurately ... Read more

August 20, 2021August 20, 2021

Yellow Tongue Leads to Rare CAD Diagnosis

Cold Agglutinin News

A young boy’s bright yellow tongue led doctors to a diagnosis of cold agglutinin disease (CAD), as described in a report of this rare case. The report, “An Icteric Tongue,” ... Read more

August 19, 2021August 20, 2021

Oxford-Harrington Partnership Targets FA as First Priority

Friedreich's Ataxia News

Scientists on either side of the Atlantic have teamed up, through the new Oxford-Harrington Rare Disease Centre (OHC), to address unmet needs in rare disease research and deliver “major clinical ... Read more

August 17, 2021August 17, 2021

GI Symptoms in LOPD Widespread, Study Finds

Pompe Disease News

More attention should be given to evaluating the gastrointestinal symptoms of individuals with late-onset Pompe disease (LOPD) because they cause a significant disease burden, according to a recent study. The ... Read more

August 17, 2021August 17, 2021

Teenager’s Response Supports Pitolisant, Now in Phase 2 Trial, in Easing PWS

Prader-Willi News

Pitolisant, a treatment for narcolepsy, should be considered for people with Prader-Willi syndrome (PWS) whose response to psychotropic medications is less than optimal, a case report of a 15-year-old patient ... Read more

August 17, 2021August 17, 2021

Lung Transplant Biorepository, Registry Targets Patient Outcomes

Cystic Fibrosis News Today

The Cystic Fibrosis Foundation (CFF) and the Cleveland Clinic are launching the Cystic Fibrosis Lung Transplant Consortium (CFLTC) Biorepository and Registry, aimed at supporting research into understanding the factors that ... Read more

August 16, 2021August 16, 2021

#AAIC21 – BIIB080 Safely Lowered Tau Levels in Patients in Phase 1 Trial

Alzheimer's News Today

BIIB080, an investigational therapy for Alzheimer’s disease, was safe and well-tolerated, and sustainably lowered patients’ levels of tau — a protein linked to neurodegeneration, according to topline results of a ... Read more

August 16, 2021August 16, 2021

‘Personal Zen’ Mobile App Helps to Ease Anxiety in Pediatric-onset MS

Multiple Sclerosis News Today

Personal Zen, a digital anxiety management tool developed by Wise Therapeutics, eased anxiety and broad negative moods in people with pediatric-onset multiple sclerosis (MS), according to the results of a pilot ... Read more

August 16, 2021August 17, 2021

Interactive Research, Rehab Center for DBS Opening at Mount Sinai

Parkinson's News Today

People with Parkinson’s disease who undergo deep brain stimulation (DBS) at Mount Sinai in New York, along with those scheduled for the surgical treatment, will soon have access to Q-Lab, ... Read more

August 16, 2021August 16, 2021

Food & Friends, CFF Partner to Deliver Tailored Meals to Patients

Cystic Fibrosis News Today

Food & Friends will be delivering medically tailored meals to people living with cystic fibrosis (CF) in the Washington, D.C., area, as part of a new collaboration with the Cystic ... Read more

August 13, 2021August 13, 2021

Prime Editing, Advance on CRISPR, Shows Potential on CFTR Mutations

Cystic Fibrosis News Today

Researchers corrected mutations underlying cystic fibrosis (CF) in a three-dimensional (3D) cell model of the disorder, using a new form of gene editing. This work serves as a proof-of-principle for ... Read more

August 12, 2021August 12, 2021

Twins’ Different Symptoms Complicate Diagnosis

Batten Disease News

Juvenile Batten disease is difficult to diagnose promptly and accurately, as demonstrated by the case of twin girls with different symptoms of the same disorder. The case suggests that a ... Read more

August 12, 2021August 12, 2021

CTRP9 Protein Levels May Be Biomarker of Lung Disease Severity

Scleroderma News

High levels of a protein known as CTRP9 appear to indicate worsening lung function in patients with systemic scleroderma (SSc), according to a recent study. The discovery suggests that CTRP9 ... Read more