article thumbnail

Researchers design genetic therapy to prevent brain deterioration

Drug Discovery World

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. Doctors believed the calcium deposits were non-specific signs of brain damage, but this research challenges this view.

Therapies 147
article thumbnail

Combination therapy halts lung cancer tumour growth in mice

Drug Discovery World

A pathway to better therapies. The findings suggest a pathway to better therapies for one of the least treatable forms of cancer. One of the challenges we will face is convincing doctors to go back to an old drug. But the nice thing about this strategy is that it may work where current therapies have failed.

Therapies 130
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Sloane’s Journey with ASO Therapy

KIF1A

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. March 2024 – The FDA approved the IND, and the administering doctor (Dr. Hello KIF1A Community!

article thumbnail

UK’s NICE supports CAR-T for young people with leukaemia

Drug Discovery World

The treatment becomes only the second CAR-T cell therapy to secure routine funding for use in the NHS, and the only one for treating ALL in children and young adults under 26 years old. It offers a chance of durable remissions and prolonged overall survival for people who often have no other option.

Doctors 130
article thumbnail

Is There a Better Therapy for Hospitalized Anorexia Patients?

The Pharma Data

20, 2020 — It may seem counterintuitive, but when someone with the eating disorder anorexia nervosa is hospitalized, treatment often begins by cutting calories. Now, new research suggests that those eating restrictions can be safely relaxed in the hospital. That led to shorter hospital stays, the study found. TUESDAY, Oct.

article thumbnail

Sloane’s 1st ASO treatment

KIF1A

They prepared for this first dose with numerous assessments and evaluations, including a neurological exam, overnight EEG, cognitive evaluation, physical therapy assessment, blood work, and data collection using a movement monitor. On the day of the treatment, they checked into the hospital where Sloane was administered sedation medication.

article thumbnail

Basildon University Hospital maternity unit rated 'inadequate'

The Pharma Data

Basildon University Hospital has been told its maternity services were inadequate after a whistleblower’s fears sparked a surprise inspection. A Care Quality Commission (CQC) report rated the unit at Basildon University Hospital as inadequate with “failings” found in six other serious cases. Image copyright.