Posts by: Aisha I Abdullah PhD

A 47-year-old woman with a newly described COL7A1 gene mutation was diagnosed with dystrophic epidermolysis bullosa (DEB), and possibly an unusual subtype affecting the extremities. She had a history of ... Read more

June 16, 2021June 16, 2021

Soliris Helps Treat aHUS Relapse with Kidney Disease During Pandemic

aHUS News, COVID-19 News

Soliris (eculizumab) is effective for treating relapses in patients with atypical hemolytic uremic syndrome, even in the presence of severe kidney disease, according to a case report. The case study ... Read more

June 15, 2021June 15, 2021

Galyan Bio Plans to Start Clinical Testing of Oral Therapy in 2022

Huntington's Disease News

Galyan Bio plans to initiate a clinical trial of a small molecules candidate that aims to slow the progression of Huntington’s disease next year, working in with Bicoll GmbH. Galyan Bio is developing and optimizing ... Read more

June 14, 2021June 15, 2021

Lung Cancer More Common in COPD Patients, Japanese Study Finds

COPD News Today

Although lung cancer development is unpredictable, patients with chronic obstructive pulmonary disease (COPD) appear more likely to have the cancer and a worse prognosis compared with the general population, a ... Read more

June 14, 2021June 14, 2021

Tau Levels in Spinal Fluid Possible Biomarker of ALS, Likely Progression

ALS News Today

Levels of the protein tau found in the cerebrospinal fluid (CSF) — the transparent liquid surrounding the brain and spinal cord — can help in diagnosing amyotrophic lateral sclerosis (ALS), a ... Read more

June 11, 2021June 11, 2021

CFTR Modulator Adherence High in Kids, Teens on Symdeko, Orkambi

Cystic Fibrosis News Today

Children and adolescents on Symdeko (tezacaftor/ivacaftor) and Orkambi (ivacaftor/lumacaftor) for cystic fibrosis (CF) had higher treatment adherence rates compared with adults given the same CFTR modulators, a nationwide study in the U.S. ... Read more

June 11, 2021June 11, 2021

Man With Hemophilia A Treated for COVID-19 While on Hemlibra

COVID-19 News, Hemophilia News Today

A 70-year-old man with severe hemophilia A was treated for COVID-19 while receiving prophylactic, or preventive, treatment with Hemlibra (emicizumab) to lower his bleeding risk. This case underscores the importance ... Read more

June 10, 2021June 10, 2021

Inflammatory Immune Cells Found in Active, But Not Chronic, Brain Lesions

Multiple Sclerosis News Today

A class of immune cells called myeloid cells that express the inflammatory protein SIGLEC1 may be able to distinguish between active and chronic multiple sclerosis (MS) brain lesions, a study ... Read more

June 8, 2021June 8, 2021

Genetic Cause of PWS Can Affect Age at Diagnosis, Therapy Start

Prader-Willi News

The genetic profile of children with Prader-Willi syndrome (PWS) correlates with their age at diagnosis and at the start of growth hormone treatment, as well as with pre-treatment levels of insulin-like ... Read more

June 3, 2021June 3, 2021

Study Urges Close Monitoring of Patients Starting Off-label Takhzyro

Angioedema News

A hereditary angioedema (HAE) patient with a rare gene mutation underwent several rounds of treatment adjustments and experienced multiple side effects while transitioning to off-label Takhzyro (lanadelumab‐flyo) over a period ... Read more

June 3, 2021June 3, 2021

NfL Promising Biomarker for Predicting Tecfidera Response

Multiple Sclerosis News Today

Low levels of serum neurofilament light chains (sNfL) prior to treatment predicts an optimal response to Tecfidera (dimethyl fumarate) and reduced immune cell overactivity in multiple sclerosis (MS) patients, a ... Read more

June 2, 2021June 2, 2021

High Cortisol Levels Seen to Ease Psoriasis in Cyclic Cushing’s Patient

Cushing's Disease News

Symptoms of psoriasis vulgaris, an autoimmune disease that mainly affects the skin, eased in a man with cyclic Cushing’s disease during a short episode of hypercortisolemia, a period marked by cortisol levels ... Read more

May 26, 2021May 27, 2021

Psoriasis Drug Soriatane Eases Bone Defects in Zebrafish CEP Model

Porphyria News

Soriatane (acitretin), a medication normally used to treat the skin disorder psoriasis, was found to alleviate bone defects in a new zebrafish model of congenital erythropoietic porphyria, or CEP, a ... Read more

May 26, 2021May 26, 2021

Race, Income Limit CF Lung Transplant Access in US More Than Canada

Cystic Fibrosis News Today

Patients in the U.S. with cystic fibrosis (CF) and advanced lung disease are less likely to receive a lung transplant and twice as likely to die before having transplant surgery ... Read more

May 26, 2021May 26, 2021

MPZ Deficiency Mutations Linked to Milder Neuropathy in CMT1B

Charcot-Marie-Tooth News

Mild sensory neuropathy was found in a group of Charcot-Marie-Tooth disease type 1 subtype B (CMT1B) patients with myelin protein zero (MPZ) gene mutations that cause insufficient levels of MPZ ... Read more