Posts by: Patricia Inacio PhD

aHUS News

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate ... Read more

August 3, 2022August 3, 2022

Kaftrio Boosts Exercise Capacity in Teens With CF, Case Analysis Shows

Cystic Fibrosis News Today

A six-week treatment with Kaftrio led to marked improvement in exercise capacity in adolescents with cystic fibrosis (CF), according to an analysis of three cases. The study, “Elexacaftor–Tezacaftor–Ivacaftor improves exercise capacity ... Read more

July 27, 2022July 27, 2022

Soliris Briefly Used to Safely Treat Severe aHUS Episode in Teen With SLE

aHUS News

Soliris (eculizumab) safely and effectively treated a severe episode of atypical hemolytic uremic syndrome (aHUS) in an adolescent with another autoimmune disease called systemic lupus erythematosus (SLE), a case report described. The ... Read more

July 8, 2022July 8, 2022

Childhood Medical Trauma Reported by Most Adults With CF in UK Study

Cystic Fibrosis News Today

Most adults with cystic fibrosis (CF) report experiences with painful or frightening medical procedures in childhood after their diagnosis, according to a U.K. study. These patients may benefit from psychosocial ... Read more

June 28, 2022June 28, 2022

Dosing Begins in Phase 1/2 Trial of BX004 for P. aeruginosa Infections

Cystic Fibrosis News Today

Two patients have been dosed in a clinical trial testing BX004, a nebulized phage therapy designed to kill Pseudomonas aeruginosa, the most common cause of lung infections in people with cystic fibrosis (CF). The ... Read more

June 20, 2022June 20, 2022

Gut Microbiome of Patients Favors Inflammation, Study Suggests

Parkinson's News Today

People with Parkinson’s disease appear to have a gut microbiome — the population of microorganisms living in the intestines — that is shifted toward a pro-inflammatory state, a small study ... Read more

June 17, 2022June 17, 2022

Foot Deformity Doesn’t Affect Walking Impairments in CMT: Study

Charcot-Marie-Tooth News

The ability to walk in children and adolescents with Charcot-Marie-Tooth (CMT) disease is similarly impaired regardless of their type of foot deformity, according to a small study. The study, “Do ... Read more

June 15, 2022June 15, 2022

Transsphenoidal Surgery May Be Safe for Older Adults, Study Shows

Cushing's Disease News

Transsphenoidal surgery (TSS), a minimally invasive surgery to remove tumors from the brain’s pituitary gland, was deemed safe and effective to treat Cushing’s disease in a 67-year-old woman, as described ... Read more

June 14, 2022June 15, 2022

Function of Regulatory T-cells Impaired in Women With Sjögren’s: Study

Sjogren's Syndrome News

The function of immune regulatory T-cells (Tregs) — cells that play a key role in dampening the body’s immune response — is impaired in women with Sjögren’s syndrome, a study ... Read more

June 14, 2022June 14, 2022

New Human Cell Model From Bit.Bio Aims to Advance Research, Therapies

Huntington's Disease News

Bit.Bio has launched a new human cell model to advance research and accelerate therapeutic development for Huntington’s disease. The cells, called ioGlutamatergic Neurons HTT-50CAG/T, are engineered to carry a specific mutation ... Read more

June 14, 2022June 14, 2022

MG Symptoms Weigh on Life Quality, Particularly for Women

Myasthenia Gravis News

People with myasthenia gravis, particularly women, have a high symptom burden that considerably affects their health-related quality of life, but the disease’s human cost is rarely given adequate study, a systematic ... Read more

June 13, 2022June 13, 2022

Study Reveals Risk Factors for Future Bleeds in Hemophilia Patients

Hemophilia News Today

The levels of blood clotting factors, physical activity, and bleeding history are all potential risk factors for future bleeds in hemophilia patients under preventive therapy, a small review study from ... Read more

June 13, 2022June 13, 2022

Genetic Testing May Identify New Gene Mutations in EDS: Study

Ehlers-Danlos News

A genetic testing technique called next-generation sequencing (NGS) may help unveil unrecognized disease-causing mutations linked with Ehlers-Danlos syndrome (EDS) and related disorders, a Polish study suggests. The study “Next-Generation Sequencing ... Read more

June 13, 2022June 13, 2022

KalVista Presents Positive Data of Sebetralstat and Oral KV998086

Angioedema News

Sebetralstat, KalVista Pharmaceuticals‘ investigational on-demand treatment for swelling attacks in people with hereditary angioedema (HAE), led to a rapid and sustained suppression of kallikrein in HAE patients, according to data ... Read more

June 9, 2022June 10, 2022

FibroGen Completes Enrollment for Second Phase 3 Trial of Pamrevlumab

Muscular Dystrophy News

FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD). The LELANTOS-2 study (NCT04632940) ... Read more