Posts by: Aisha I Abdullah PhD

A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to educational resources for students of color and economically disadvantaged youngsters with intellectual and developmental disabilities ... Read more

March 4, 2021March 5, 2021

Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited

COVID-19 News, Muscular Dystrophy News

The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b clinical trial, assessing the anti-inflammatory steroid therapy vamorolone in boys with Duchenne muscular dystrophy (DMD). ... Read more

March 2, 2021March 2, 2021

Potential IPF Treatment, Found Using AI, May Move Into Trial, Insilico Says

Pulmonary Fibrosis News

Insilico Medicine announced that it has found a new therapeutic target and potential therapy candidate for idiopathic pulmonary fibrosis (IPF) using artificial intelligence (AI), a company specialty. The potential therapy, a ... Read more

February 26, 2021February 26, 2021

Generalized MG Patients Needed for UCB’s Global Rozanolixizumab Trial

Myasthenia Gravis News

An international Phase 3 clinical trial assessing the efficacy, safety, and tolerability of rozanolixizumab as a treatment for generalized myasthenia gravis (MG) is currently recruiting participants at 114 study locations. ... Read more

February 24, 2021February 24, 2021

Early ERT Key in Severe Classic Infantile Pompe

Pompe Disease News

Early treatment with enzyme replacement therapy (ERT) and immune tolerance induction (ITI) greatly improved clinical outcomes for infants with the severe, frequently ERT-nonresponsive, CRIM-negative infantile Pompe disease (IPD), a study has found. ... Read more

February 24, 2021February 24, 2021

COVID Reinfection Possibly Due to Immunosuppressant Use for AAV

ANCA Vasculitis News, COVID-19 News

A woman was potentially reinfected with SARS-CoV-2 — the virus that causes COVID-19 — while on immunosuppressive therapy for ANCA-associated vasculitis (AAV). This case suggests that immunosuppressed patients are at risk ... Read more

February 24, 2021February 25, 2021

Survey Highlights Need to Align Adult PKU Treatment to Improve Patient Outcomes

Phenylketonuria News

A new survey of six Italian clinics found varying and inconsistent approaches for managing adult phenylketonuria (PKU), with insufficient PKU diet therapy and poor patient adherence to such eating plans. ... Read more

February 24, 2021February 24, 2021

MJFF Backs Investigation of Parkinson’s-linked Protein Complex

Parkinson's News Today

A project investigating a complex formed by two key proteins involved in Parkinson’s disease development has received nearly $150,000 in funding from the Michael J. Fox Foundation for Parkinson’s Research ... Read more

February 23, 2021February 23, 2021

PWS Subtypes May Respond Differently to Psychotropic Treatments

Prader-Willi News

A small study exploring gene alterations related to Prader-Willi syndrome (PWS) suggests that subtypes of the disorder have different genetic profiles that may affect response to psychotropic treatments and potential ... Read more

February 23, 2021February 23, 2021

Study Identifies Genetic Risk, Protective Factors for Late-onset MG in Italy

Myasthenia Gravis News

A specific haplotype — or genetic signature inherited from one parent — was identified as a risk factor for the development of generalized late-onset myasthenia gravis (LOMG) in the Italian ... Read more

February 23, 2021February 24, 2021

Focus Groups Highlight Need for Patient Input in Diagnosis, Treatment

FAP News Today

In focus groups, transthyretin (ATTR) amyloidosis patients and families reported challenges and concerns related to the odyssey of getting a diagnosis, the effect of symptoms on quality of life, and ... Read more

February 22, 2021February 22, 2021

Case Highlights Unusual Respiratory Features in Severe Bardet-Biedl Syndrome

Genetic Obesity News

A case study of a baby with Bardet-Biedl syndrome (BBS) highlights the unusual respiratory features that result from inheriting two mutant copies of the BBS10 gene. The report, “Bardet-Biedl syndrome ... Read more

February 19, 2021February 19, 2021

DiamiR Receives Patent Allowance for Rett MicroRNA Biomarker

Rett Syndrome News

The United States Patent and Trademark Office (USPTO) has issued an allowance for a patent submitted by DiamiR for methods the company developed that use microRNA (miRNA) as biomarkers of ... Read more

February 19, 2021February 19, 2021

Gene Therapy Seen to Rescue Motor, Nerve Defects in CMTX1 Mouse Model

Charcot-Marie-Tooth News

Delivering a healthy neurotrophin-3 (NT-3) gene to muscle cells in a mouse model of Charcot-Marie-Tooth disease type X1 (CMTX1) prevented motor function decline and eased defects in nerve and muscle ... Read more

February 18, 2021February 18, 2021

Poor Core Stability Evident in SSc Patients, Affecting Life Quality

Scleroderma News

Patients with scleroderma have poor core stability, as measured by trunk muscle strength and endurance, affecting both their physical capacity and daily performance, a Turkish study found. Previous studies largely ... Read more