For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Amicus Therapeutics (FOLD) remains a Buy, driven by strong product momentum and new assets. Read here for more investment ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.

Alector Therapeutics is shelving a GSK-partnered antibody after the asset failed to slow disease progression in patients with ...

The Batten Disease pipeline presents opportunities with over 10 companies developing 12+ drugs in varied clinical stages. Novel approaches, including gene therapy and enzyme replacement, address unmet ...

Australians with the extremely rare and life-threatening inherited disease, acid sphingomyelinase deficiency (ASMD) will now have free access to a ...

This represents Alector's second failed neurodegenerative asset in a year, after an AbbVie-partnered asset missed in ...

The Israel-based company, which developed Elfabrio alongside Chiesi Global Rare Diseases, part of Italy-based Chiesi Group, had been seeking approval for a new, less frequent dosing regimen for ...

(Nasdaq: ALEC), a clinical-stage biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, today announced results from the Phase 3 ...

About Chiesi Group Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty ...

Pancreatitis can cause nutrient deficiencies. A well-balanced diet can help relieve symptoms such as inflammation and pain, as well as improve digestion.